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Displaying 1 to 7 of 7 results for gene therapy

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  • Green Group

    The Green Group is the biomaterials and drug delivery laboratory in the Biomedical Engineering Department at the Johns Hopkins University School of Medicine. Our broad research interests are in cellular engineering and in nanobiotechnology. We are particularly interested in biomaterials, controlled drug delivery, stem cells, gene therapy, and immunobioengineering. We are working on the chemistry/biology/engineering interface to answer fundamental scientific questions and create innovative technologies and therapeutics that can directly benefit human health.

    Research Areas: nanobiotechnology, stem cells, biomedical engineering, drugs, immunobioengineering

    Lab Website

    Principal Investigator

    Jordan Green, Ph.D.

    Department

    Biomedical Engineering

  • Haig Kazazian Lab

    The Kazazian Lab focuses on the biology of LINE-1 (L1) retrotransposons. Retrotransposons are pieces of genomic DNA that have the ability to duplicate themselves and insert into a new genomic location. Current studies use innovative DNA sequencing to locate all human-specific L1s in any genome. By understanding L1 biology, we hope to better understand the role of these genomes and their behavior in complex human disease, such as cancer and mental disorders. The lab is also examining how to carry out gene therapy of hemophilia A using AAV vectors.

    Research Areas: cell biology, cancer, retrotransposons, DNA, genomics, mental disorders

    Lab Website

    Principal Investigator

    Haig Kazazian, M.D.

    Department

    Pediatrics

  • Liudmila Cebotaru Lab

    Research in the Liudmila Cebotaru Lab studies cystic fibrosis transmembrane conductance regulator (CFTR) mutants. We also investigate corrector molecules that are currently in clinical trials to get a better understanding of their mechanism of action. A major focus of our research is on developing more efficient gene therapy vectors with the ultimate goal of developing a gene therapy for cystic fibrosis.

    Research Areas: cell biology, cystic fibrosis, kidney diseases, gene therapy, corrector molecules

    Principal Investigator

    Liudmila Cebotaru, J.D., M.D.

    Department

    Medicine

  • Retinal Cell and Molecular Lab

    The Retinal Cell and Molecular Laboratory has three major areas of interest, each of which deals with some aspect of growth factor signaling and function in the retina and retinal pigmented epithelium (RPE): 1. Investigations aimed at gaining a better understanding of the pathogenesis of retinal and choroidal neovascularization and developing new ways to treat them.
    2. Investigations aimed at understanding the molecular signals involved in retinal and RPE wound repair and scarring. The prototypical disease in this category is proliferative vitreoretinopathy and our laboratory is seeking to identify new treatments for it. 3. Investigations aimed at understanding why retinal degenerations occur and how they might be treated, with particular emphasis on neurotrophic factors.

    Research Areas: choroidal neovascularization, branch retinal vein occlusion, central retinal vein occlusion, diabetic macular edema, gene therapy, ranibizumab

    Lab Website

    Principal Investigator

    Peter Campochiaro, M.D.

    Department

    Ophthalmology

  • Vestibular NeuroEngineering Lab

    Research in the Vestibular NeuroEngineering Lab (VNEL) focuses on restoring inner ear function through “bionic” electrical stimulation, inner ear gene therapy, and enhancing the central nervous system’s ability to learn ways to use sensory input from a damaged inner ear. VNEL research involves basic and applied neurophysiology, biomedical engineering, clinical investigation and population-based epidemiologic studies. We employ techniques including single-unit electrophysiologic recording; histologic examination; 3-D video-oculography and magnetic scleral search coil measurements of eye movements; microCT; micro MRI; and finite element analysis. Our research subjects include computer models, circuits, animals and humans. For more information about VNEL, click here.
    VNEL is currently recruiting subjects for two first-in-human clinical trials:
    1) The MVI Multichannel Vestibular Implant Trial involves implantation of a “bionic” inner ear stimulator intended to partially restore sensation... of head movement. Without that sensation, the brain’s image- and posture-stabilizing reflexes fail, so affected individuals suffer difficulty with blurry vision, unsteady walking, chronic dizziness, mental fogginess and a high risk of falling. Based on designs developed and tested successfully in animals over the past the past 15 years at VNEL, the system used in this trial is very similar to a cochlear implant (in fact, future versions could include cochlear electrodes for use in patients who also have hearing loss). Instead of a microphone and cochlear electrodes, it uses gyroscopes to sense head movement, and its electrodes are implanted in the vestibular labyrinth. For more information on the MVI trial, click here.
    2) The CGF166 Inner Ear Gene Therapy Trial involves inner ear injection of a genetically engineered DNA sequence intended to restore hearing and balance sensation by creating new sensory cells (called “hair cells”). Performed at VNEL with the support of Novartis and through a collaboration with the University of Kansas and Columbia University, this is the world’s first trial of inner ear gene therapy in human subjects. Individuals with severe or profound hearing loss in both ears are invited to participate. For more information on the CGF166 trial, click here.
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    Research Areas: neuroengineering, audiology, multichannel vestibular prosthesis, balance disorders, balance, vestibular, prosthetics, cochlea, vestibular implant

  • William B. Guggino Lab

    Work in the William B. Guggino Lab focuses on the structure of the cystic fibrosis transmembrane conductance regulator (CFTR) and water channels; the molecular structure of transport proteins in epithelial cell membranes; and gene therapies to treat cystic fibrosis (CF) patients. We are also working to identify CF’s specific defect in chloride channel regulation. One recent study showed that insulin-like growth factor 1 (IGF-1) enhances the protein expression of CFTR.

    Research Areas: cell biology, cystic fibrosis, kidney diseases, gene therapy, ion channels

    Lab Website

    Principal Investigator

    William Guggino, Ph.D.

    Department

    Physiology

  • Zack Wang Lab

    The Wang lab focuses on the signals that direct the differentiation of pluripotent stem cells, such as induced-pluripotent stem (iPS) cells, into hematopoietic and cardiovascular cells. Pluripotent stem cells hold great potential for regenerative medicine. Defining the molecular links between differentiation outcomes will provide important information for designing rational methods of stem cell manipulation.

    Research Areas: pluripotent stem cells, stem cells, molecular genetics, stem cell biology, gene therapy

    Principal Investigator

    Zack Wang, Ph.D.

    Department

    Medicine

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