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      Adeno-Associated Virus Vector for Central Nervous System Gene Therapy.

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          Abstract

          The past several years have witnessed significant advances in the development of therapeutic gene delivery for neurological disorders of the central nervous system (CNS). In particular, genome-wide sequencing analysis has deepened our understanding of mutations that underlie many monogenic disorders, which in turn has contributed to clinical advances involving adeno-associated virus (AAV) vector delivery of replacement genes to treat recessive disorders. Moreover, gene therapy has been further bolstered with advances in genome editing tools that allow researchers to silence, repair, and amend endogenous genes. However, despite strong preclinical and clinical progress, challenges remain, including delivery and safety. Here, we discuss advances in AAV engineering, recent developments in cargo design, and translation of these technologies towards clinical progress.

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          Author and article information

          Journal
          Trends Mol Med
          Trends in molecular medicine
          Elsevier BV
          1471-499X
          1471-4914
          June 2021
          : 27
          : 6
          Affiliations
          [1 ] California Institute for Quantitative Biosciences, University of California, Berkeley, CA, 94720, USA.
          [2 ] Department of Molecular and Cell Biology, University of California, Berkeley, CA, 94720, USA.
          [3 ] Department of Bioengineering, University of California, Berkeley, CA, 94720, USA.
          [4 ] California Institute for Quantitative Biosciences, University of California, Berkeley, CA, 94720, USA; Department of Molecular and Cell Biology, University of California, Berkeley, CA, 94720, USA; Department of Bioengineering, University of California, Berkeley, CA, 94720, USA; Department of Chemical and Biomolecular Engineering, University of California, Berkeley, CA, 94720, USA; Helen Wills Neuroscience Institute, University of California, Berkeley, CA, 94720, USA. Electronic address: schaffer@berkeley.edu.
          Article
          S1471-4914(21)00095-2
          10.1016/j.molmed.2021.03.010
          33895085
          9113c8e5-454e-4f4d-b415-6dfe1837c9d5
          History

          gene therapy,AAV,CNS
          gene therapy, AAV, CNS

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