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Department:
Infectious Diseases and Immunology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
Yes
Last Updated
6/15/2020
Research Description
Cellular and molecular bases of host-parasite interactions; immunology of hemoparasitic diseases.
Department:
Anatomy & Cell Biology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/15/2020
Research Description
Graduate and professional student education are the main focus of my faculty activities.
Department:
Ophthalmology
Concentrations:
Accepting Students
Yes
Accepting Students if
Funding Obtained
Yes
Last Updated
3/17/2020
Research Description
Research is focused on mechanisms of inherited retinal degeneration and neuroprotection. His most recent studies have shown the important contribution of endogenously expressed cytokines such as leukemia inhibitory factor (LIF)
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/16/2020
Research Description
Functional genomics; gene expression profiling using DNA microarrays to understand phenotype.Developing gene expression classifiers of prognostic value.
Department:
Pediatrics
Concentrations:
Accepting Students
Yes
Accepting Students if
Funding Obtained
No
Last Updated
6/16/2020
Research Description
Sumita Bhaduri-McIntosh is a physician-scientist whose research bridges the fields of Virology, Oncology and Immunology. Research in the Bhaduri laboratory is focused on discovering fundamental biological pathways and understanding cancer development and progression by studying the interaction between Epstein-Barr virus (EBV; a cancer-causing herpesvirus) and its host, the B cell, by investigating two main areas: 1) investigating how EBV subverts anti-pathogen and anti-cancer barriers such as immune responses and the DNA-damage response (DDR) to drive B cell proliferation and transformation, and 2) identifying host factors that determine susceptibility of EBV-infected B cells to lytic activation, a process important for herpesvirus pathology and persistence in humans, and for lymphomagenesis.
Department:
Biochemistry & Molecular Biology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
Yes
Last Updated
6/15/2020
Research Description
Biochemistry of DNA replication and repair; dynamic protein DNA interactions and enzyme mechanisms required for these processes.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
Yes
Last Updated
6/16/2020
Research Description
Molecular pathogenesis of the herpesviruses; role of long non-coding RNAs and miRNAs on latency and reactivation; development of novel therapeutics to disrupt HSV latency and block recurrent disease.
Department:
Neuroscience
Concentrations:
Accepting Students
Yes
Accepting Students if
Funding Obtained
No
Last Updated
6/16/2020
Research Description
My lab uses transgenic & knockout mouse models, and cell models, to determine molecular mechanisms by which mutations in specific genes cause familial neurodegenerative diseases - primarily Alzheimer's disease and amyotrophic lateral sclerosis.
Department:
Pediatrics
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/16/2020
Research Description
Optimizing gene therapy vectors (AAV) for the treatment of inherited retinal disease.
Department:
Medicine
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/15/2020
Research Description
My lab studies the response of blood vessels within the lung to low oxygen levels, and how this may contribute to the development of high blood pressure in the lungs (pulmonary hypertension), and heart failure, in patients with chronic lung diseases. We do so primarily through examination of how certain types of circulating myeloid-derived cells confer increased risk to development of disease. Our goal is to discover molecular targets for drug discovery in this specific type of pulmonary hypertension.
Department:
Biochemistry & Molecular Biology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
Yes
Last Updated
6/16/2020
Research Description
Analysis of long distance gene regulation by enhancers and locus control region. Genomic and proteomic analysis of erythroid transcription factors. Modulation of gene expression using synthetic zinc finger proteins.
Department:
Pediatrics
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/15/2020
Research Description
Cardiovascular gene therapy; adeno-associated viral vectors.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
Yes
Last Updated
6/20/2019
Research Description
Developmental Biology & Evolution: Embryonic development of genitourinary organs; Genetic & genomic mechanisms of animal evolution; Genetic and environmental causes of birth defects
Department:
Pathology, Immunology & Laboratory Medicine
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/23/2020
Research Description
Research focuses on complex genetic disorders and gene-environment interactions, and includes studies of radiation sensitivity, breast cancer, and type 1 diabetes
Department:
Pathology, Immunology & Laboratory Medicine
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
7/6/2017
Research Description
Innate and acquired immune mechanisms in the pathogenesis of kidney disease: chronic allograft mephropathy, diabetes and lupus nephritis.
Department:
Infectious Diseases and Pathology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/28/2018
Research Description
Design, construction and evaluation of vaccines for companion and farm animals and humans to prevent bacterial, viral and parasite infections, confer infertility and treat tumors.
Department:
Ophthalmology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/16/2020
Research Description
Dr. Dinculescu received her Ph.D. in Biochemistry from University of Florida in 2002. Her research is focused on understanding the molecular mechanisms in several retinal degeneration disorders, including Usher syndrome. The ultimate goal is to develop therapeutic strategies to prevent the loss of sight caused by these disorders. Usher syndrome type III (USH3A), an autosomal recessive disorder, is caused by mutations in Clarin-1 (CLRN1) protein, leading to combined vision and hearing loss. The main goal of this project is to understand the function of CLRN1 in the retina, identify its binding partners, and its cellular localization pattern. This information will be used to develop a rational treatment for the USH3A disorder. She is also interested in protein aggregation mechanisms leading to the formation of extracellular deposits associated with vision loss in age-related macular degeneration, a major cause of blindness in the elderly population.
Department:
Pediatrics
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/16/2020
Research Description
The phenomenon of genomic imprinting is the differential modification of the maternal and paternal genetic contributions to the zygote, resulting in the differential expression of parental alleles during development and in the adult. A disturbance in genomic imprinting has been found to cause several human diseases including Prader-Willi and Angelman syndromes.
Department:
Surgery
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
Yes
Last Updated
6/23/2020
Research Description
Dr. Efron has a professional interest in surgical critical care and trauma, both clinically as well as in the laboratory. He conducts basic science and translational research in sepsis and inflammation. He is currently one of the principal investigators for an NIH-funded P50 center grant for the UF Sepsis and Critical Illness Research Center to study the persistent inflammation, immunosuppression and catabolism syndrome (PICS) following sepsis in surgical intensive care unit patients. He also has his own NIH R01 funding regarding examining the hematopoietic response of the elderly to severe trauma. He was mentored by and now collaborates with Drs. Moldawer, Baker and Leeuwenburgh.
Department:
Pediatrics
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/23/2020
Research Description
Effect of immunosuppression on anti-GAA antibody development in patients with Pompe disease, clinical trial studies as part of CARRA (Childhood Arthritis and Rheumatology Research Alliance)
Department:
Neurology
Concentrations:
Accepting Students
Yes
Accepting Students if
Funding Obtained
Yes
Last Updated
6/15/2020
Research Description
Dr. Farrer's research is to predict and prevent Parkinson’s disease through a combination of human genetic analyses, molecular genetic engineering and neuronal biology, mouse modeling, brain slice electrophysiology and pharmacology. He directs the Parkinson’s Research Laboratory. Dr. Farrer also directs the Clinical Genomics Program for UF Health that seeks to improve health outcomes for patients by generating and interrogating genomic data, to accelerate diagnosis and target treatments more appropriately.
Department:
Neuroscience
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/11/2018 9:33:40 AM
Research Description
Brain aging from the epigenetic and physiological level to cognitive level. Students acquire skills in molecular or electrophysiological techniques to examination the influence of environmental and lifestyle factors on epigenetic regulation of gene transcription and synaptic plasticity mechanisms involved in memory.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
Yes
Accepting Students if
Funding Obtained
Yes
Last Updated
1/20/2021
Research Description
Dr. Fujii’s research is centered on understanding of the gene regulation focusing on the step of mRNA translation. Our research program will unravel new layers of spatiotemporal specificity and fidelity of protein synthesis, to further understand the impact of translational regulation in development and in diseases, particularly those caused by protein aggregation such as neurodegenerative disorders.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
11/20/2020
Research Description
Dr. Fujii's research is centered on understanding of the gene regulation, focusing on the step of mRNA translation. His research program will unravel new layers of spatiotemporal specificity and fidelity of protein synthesis, to further understand the impact of translational regulation in development and in diseases, particularly those caused by protein aggregation such as neurodegenerative disorders. The research vision of Dr. Fujii is to molecularly understand mammalian development and homeostasis by utilizing a variety of interdisciplinary approaches from cutting edge biochemistry to in vivo animal models.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/15/2020
Research Description
We use molecular genetics and genomics coupled with animal models to examine how a normal intestinal bacterium, Oxalobacter formigenes, helps prevent kidney stones.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/19/2019 1:17:46 PM
Research Description
Molecular pathways of embryonic development; vertebrate model systems of limb development; invertebral disk formation & disease using mouse models.
Department:
Ophthalmology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/24/2019 12:20:14 PM
Research Description
Gene expression, and gene delivery, gene therapy and functional genomics in the retina.
Department:
Medicine
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/16/2020
Research Description
Dr. Heldermon's primary focus is on developing new therapeutic strategies using gene therapy and stem cell therapy for patients with inherited diseases such as lysosomal storage disorders and hemophilias. He is focused on studying the frequent cell transfer between mother and fetus during gestation that establish microchimerism and is determining ways to modulate the level of this chimerism to enable therapy of disease. Through the use of gene replacement, he hopes to correct the underlying deficiencies underpinning the disease manifestations. • Additionally he is studying the spread of breast malignancies through the use of xenografting. By studying the spread of tumors from breast cancer patients in mice, he hopes to determine if metastases is determined by tumor specific or host factors. This model will allow identification of what factors are important in tumor spread and localization. Additionally, the model will allow testing of therapeutic agents. The goal is to identify new targets for therapy to personalize oncologic therapeutics.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/16/2020
Research Description
Molecular mechanisms of host-pathogen interactions; virulence mechanism of Pseudomonas aeruginosa infection and development of antimicrobial strategies; Cellular reprogramming and genome editing
Department:
Pediatrics
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/15/2020
Research Description
Dr. Kang’s laboratory focuses on the genetics of muscular dystrophy and the pathogenesis of disease in MEGF10 myopathy. Ultimately, these studies are expected to help identify novel therapeutic strategies for these diseases.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/20/2019 4:54:16 PM
Research Description
Pathogenesis of noroviruses, innate immune responses to norovirus infection, and molecular mechanisms of norovirus replication. Mucosal immunity to, and pathogenesis, of murine norovirus infection.
Department:
Medicine
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/30/2020
Research Description
Dr. Kaye has a longstanding research interest in cancer genetics and translational clinical research. His lab studies the functional properties of a cAMP/CREB/CRTC pathway that is activated in cancer by loss of LKB1/AMPK signals in lung cancer. He also studies oncolytic virotherapy for small cell lung cancer with plans for a phase 1 clinical trial.
Department:
Biochemistry & Molecular Biology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/19/2019 9:44:16 AM
Research Description
Mammalian cell culture systems. Epigenetic regulation of transcription; chromatin structure and function; epigenetic silencing of tumor suppressor genes in cancer; single-molecule analysis of chromatin structure
Department:
Pharmacology & Therapeutics
Concentrations:
Accepting Students
Yes
Accepting Students if
Funding Obtained
Yes
Last Updated
6/16/2020
Research Description
We study how cells communicate with each other to repair injured tissues including skeletal and cardiac muscle using sophisticated mouse genetics and state of the art cell biological tools.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/15/2020
Research Description
Dr. Lewin's current research involves the development of gene and pharmacological therapies for neurodegenerative diseases of the retina and the central nervous system. The group has developed a mouse model of Age Related Macular Degeneration, a common blinding disease of the elderly.

Chen Ling, Ph.D.

Department:
Pediatrics
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/11/2018
Research Description
Dr. Ling's research focused on gene therapy of inherited diseases, such as hemophilia and sickle cell disease. Specifically, Dr. Ling studies the interaction between adeno-associated virus and its target cells, such as hepatocytes and hematopoietic stem cells.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
10/15/2020
Research Description
Approximately 15% of cancers diagnosed were attributed to carcinogenic viral infections. Upon viral infections, the host innate immune system acts as the first line of defense to prevent viral invasion or replication, while viruses also strive for survival by repressing innate immune signaling. By further understanding how innate immunity is regulated by tumor viruses, our lab aims to dissect the interactions between innate immune responses and viral tumor development. Our long term goal is to identify potential targets and strategies for viral cancer treatment. Utilizing molecular biology as well as bioinformatics tools, our lab seeks to extend our knowledge of how viral proteins regulate the cGAS-STING pathway and how to develop potential KSHV cancer therapy by targeting these viral proteins. Specifically, we are focusing on: 1) Exploring detailed mechanisms of cGAS-STING signaling regulation by KSHV viral proteins 2) Exploring the role of KSHV viral proteins on KSHV pathogenesis in vitro and in vivo 3) Screening for novel cGAS-STING pathway agonists, and testing their efficacy using PEL based xenograft mouse model
Department:
Anatomy & Cell Biology
Concentrations:
Accepting Students
Yes
Accepting Students if
Funding Obtained
Yes
Last Updated
6/15/2020
Research Description
Characterization of regulatory variants, genetic networks and molecular pathways underlying complex diseases such as adolescent idiopathic scoliosis. My research also includes studies exploring homeostasis of connective tissue and extracellular matrix homeostasis as well as deciphering the role of non-coding variant genes in human disease.
Department:
Whitney Laboratories
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
Yes
Last Updated
6/16/2020
Research Description
Director of the Whitney Laboratory for Marine Bioscience. His research focus is aimed at elucidating the cellular and molecular basis for the evolution of cell-type complexity, regenerative biology, and cellular engineering.
Department:
Environmental Health
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/15/2020
Research Description
Pathogenic mechanisms of Shigella and Chlamydia Evolution and emergence of Shiga toxin-producing Shigella flexneri Cell wall metabolism and cell division processes of Chlamydia spp. Surveillance of non-HIV sexually transmitted infections in Haiti Laboratory capacity building
Department:
Pathology, Immunology & Laboratory Medicine
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/16/2020
Research Description
education research, team science
Department:
Biomedical Engineering
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
Yes
Last Updated
6/13/2017 11:46:34 AM
Research Description
Vascular remodeling, wound healing and tissue engineering (vascular/bone/cartilage)
Department:
Pediatrics
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
11/23/2020
Research Description
My research employs proteomics, RNA-seq and capture-seq to study transcriptional networks associated with gammaherpesvirus infections, and to discover new viruses in human diseases of unknown etiology or emerging epidemiology, at the human animal or vector interface, and in the context of immunocompromise or cancer. Special emphasis is on elucidating complex molecular mechanisms of Epstein-Barr virus lytic activation, immune subversion, and cellular transformation relevant to virus-induced cancer as well as exploiting EBV to immortalize human B-cells to identify and clone antiviral antibodies.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
Yes
Accepting Students if
Funding Obtained
Yes
Last Updated
6/30/2020
Research Description
Focused on the use of statistical theory to elucidate the relationship between genotype and phenotype. Currently interested in integration of omic data.
Department:
Pathology, Immunology & Laboratory Medicine
Concentrations:
Accepting Students
Yes
Accepting Students if
Funding Obtained
Yes
Last Updated
6/15/2020
Research Description
Genetic analysis of systemic lupus erythematosus (SLE) susceptibility using mouse models, immune metabolism and use of metabolic inhibitors to treat lupus Microbiome and lupus
Department:
Whitney Laboratories
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/20/2019 11:50:20 AM
Research Description
Genomic Bases of Memory and Neuronal Evolution. Our laboratory works to characterize basic mechanisms underlying the design of nervous systems and the evolution of neuronal signaling mechanisms. The major questions are: (1) why are individual neurons so different? (2) How do learning and memory store in neuronal genomes and neural circuits? (3) How to restore memories? (4) How to make “better” neurons and brains and improve cognitive functions?
Department:
Medicine
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/15/2020
Research Description
Molecular epidemiology and transmission modeling of Vibrio cholerae in Haiti and Bangladesh.
Department:
Medicine
Concentrations:
Accepting Students
Yes
Accepting Students if
Funding Obtained
Yes
Last Updated
6/23/2020
Research Description
The overarching goal of my research program is to understand underlying mechanisms in rare disorders that impact mitochondrial function to guide therapeutic development relevant to both rare and common disorders. Using mouse models and human patient-derived, differentiated induced pluripotent stem cells we examine cardiac, neuronal, and skeletal muscle samples in search of common effectors of mitochondrial function that can be targeted using adeno-associated virus (AAV) based delivery or other treatment strategies.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
Yes
Accepting Students if
Funding Obtained
Yes
Last Updated
6/15/2020
Research Description
Uses human and mouse genetics to understand the causes and to develop therapies for genetic neurodegenerative diseases. A major focus in the lab is on diseases caused by repeat expansion mutations including amyotrophic lateral sclerosis (ALS), myotonic muscular dystrophy (DM1 and DM2) and spinocerebellar ataxias (SCA8).
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
Yes
Accepting Students if
Funding Obtained
No
Last Updated
6/16/2020
Research Description
Biology of Kaposi's sarcoma-associated herpesvirus (KSHV); role of latency-associated nuclear antigen (LANA) in transcriptional regulation, viral DNA replication, and episomal segregation in latently-infected cells; role of virus-encoded micro RNAs.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/16/2020
Research Description
Genetic imprinting mechanisms and mouse models of imprinting disorders. Epigenetic reprogramming during germ cell development.
Department:
Pediatrics
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/28/2018
Research Description
Dr. Rivkees’ laboratory is currently focusing on the role of adenosine on cardiac development and the fetal programming of adult cardiac disease via epigenetic mechanisms. In addition, we are studying developmental brain injury, focusing on white matter injury and oligodendrocyte biology.
Department:
Pathology, Immunology & Laboratory Medicine
Concentrations:
Accepting Students
Yes
Accepting Students if
Funding Obtained
Yes
Last Updated
6/15/2020
Research Description
Molecular evolution of viruses, with special emphasis on HIV/SIV and SARS-CoV-2. Development and application of new bioinformatics and phylogenetic analysis tools to investigate the molecular epidemiology of microbial pathogens, with emphasis on RNA-viruses, Vibrio cholera, and MRSA.
Department:
Pathology, Immunology & Laboratory Medicine
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/15/2020
Research Description
Prediction, immunopathogenesis, genetics and prevention and care of Type 1 diabetes.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/19/2019 9:19:26 AM
Research Description
My laboratory works on the fundamental biology of stem cells. Our goal is to design test and bring new cell based therapies to the clinic. How stem cells are involved in cancer and the creation of a supportive "cancer niche" are two primary areas of research
Department:
Whitney Laboratories
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/15/2020
Research Description
Research focus:the cellular and molecular mechanisms of development and regeneration in annelids and other closely related marine animals. Interests: patterning of embryonic stem cells, neurogenesis, regenerative biology and patterning of the germline.
Department:
Medicine
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/22/2015
Research Description
Mechanisms of autoantibody production.
Department:
Pediatrics
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
Yes
Last Updated
6/19/2019 10:07:38 AM
Research Description
For nearly four decades, Dr. Srivastava’s research has been focused on the following two parvoviruses, the non-pathogenic adeno-associated virus (AAV), and a common human pathogen, the parvovirus B19, and the development of recombinant parvovirus vectors for human gene therapy. His laboratory has made seminal contributions to the field of parvoviruses, which include: identification of cellular co-receptors for AAV as well as parvovirus B19; elucidation of various steps involved in parvovirus trafficking in the cell and nuclear transport; identification of cellular proteins involved in the regulation of AAV DNA replication and encapsidation; development of recombinant AAV and parvovirus B19 vectors; transgenic and knockout mouse models to study parvovirus-induced pathogenicity, and the use of parvovirus vectors for gene transfer and gene therapy. Parvovirus-based vectors have gained attention as a useful alternative for human gene therapy. The stable integration of the AAV genome, and the erythroid cell tropism of B19 have been exploited to construct the following two types of AAV-B19 hybrid vectors. Type I vectors allow stable integration of the viral DNA in infected cells but the viral gene expression occurs predominantly in the erythroid progenitor cells. Type II vectors allow efficient erythroid progenitor cell-specific gene delivery as well as expression of transduced genes. However, it has become increasingly clear that the full potential of the first generation of AAV vectors is unlikely to be realized. For example, AAV vectors composed of the naturally occurring capsids trigger a host immune response, especially at high doses, since specific surface-exposed amino acid residues on the naked icosahedral capsids are readily targeted by the host cell machinery. Furthermore, the AAV genome is a single-stranded DNA, which is transcriptionally-inactive, and thus negatively impacts the transduction efficiency of the first generation of AAV vectors. During the past decade, strategies have been developed in Dr. Srivastava’s laboratory that circumvent both of these problems associated with the first generation of AAV vectors. For example, specific surface-exposed amino acid residues on the AAV capsids have been modified to develop the next generation (‘NextGen’) AAV vectors that are significantly more efficient, and are less immunogenic. One of the NextGen AAV vectors has been used by other investigators in a Phase I Clinical Trial for Leber's Hereditary Optic Neuropathy (LHON), and has shown clinical efficacy. The single-stranded AAV genome has also been modified to develop generation X (‘GenX’) AAV vectors with which enhanced transgene expression can be achieved. The combination of the NextGen and the GenX vectors has resulted in the development of optimized (‘Opt’) AAV vectors that are more efficient at further reduced doses. The current emphasis is on developing recombinant parvovirus vectors for gene therapy/genome editing of genetic diseases such as beta-thalassemia and sickle cell disease, and gene therapy of malignant disorders such as hepatoblastoma and hepatocellular carcinoma.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
Yes
Last Updated
6/16/2020
Research Description
Mouse models for neurological disease;RNA processing, transport and turnover; molecular mechanisms of mammalian development and aging
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
Yes
Last Updated
6/30/2020
Research Description
Oncogenic virus pathogenesis, noncoding RNAs, virus-host interactions, host immunity to viruses, viral latency.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/15/2020
Research Description
Mutation and analysis of human disease and susceptibility genes.
Department:
Medicine
Concentrations:
Accepting Students
Yes
Accepting Students if
Funding Obtained
Yes
Last Updated
6/16/2020
Research Description
Our current research falls within the general themes of host-pathogen interactions, and is divided into two major areas 1) Molecular studies of HIV and HCV pathogenesis and drug resistance and 2) Ecology of indigenous microbial communities associated with human infections.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/1/2016
Research Description
We study microsatellite repeat expansion diseases, including myotonic dystrophy. We use molecular, cellular, computational, and animal-based approaches to understand basic mechanisms that cause symptoms in this and related multi-systemic diseases, as well as identify treatments that will help patients. We also study RNA localization at a basic level, to understand why and how RNA molecules move to different places in the cell. We seek to combine high-throughput genomics approaches with single molecule imaging approaches to characterize and understand this new frontier in biology.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
Yes
Last Updated
6/16/2020
Research Description
We are studying cancer cell signaling with expertise in signal transduction, genetic and epigenetic regulation, and animal models. Our current laboratory research is centered on two families of transcriptional co-activators, MAML and CRTC, which are essential components of Notch receptor signaling and LKB1-CRTC/CREB signaling pathways, respectively. We are investigating the molecular regulation and the roles of deregulated cell signaling in cancer generation and progression, with the goal of gaining molecular insights into cancer pathogenesis and identifying novel cancer diagnostic and therapeutic targets.
Department:
Pathology, Immunology & Laboratory Medicine
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
No
Last Updated
6/16/2020
Research Description
General Neuropathology. Neurodegenerative diseases.
Department:
Molecular Genetics & Microbiology
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
Yes
Last Updated
6/30/2020
Research Description
We are interested in understanding mechanisms mediating innate immunity against pahtogen infection as well as the dysregulation of innate immunity assoicated with aging.
Department:
Pediatrics
Concentrations:
Accepting Students
No
Accepting Students if
Funding Obtained
Yes
Last Updated
6/16/2020
Research Description
AAV vector design. AAV vector production.

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