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Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species.
Tabebordbar, Mohammadsharif; Lagerborg, Kim A; Stanton, Alexandra; King, Emily M; Ye, Simon; Tellez, Liana; Krunnfusz, Allison; Tavakoli, Sahar; Widrick, Jeffrey J; Messemer, Kathleen A; Troiano, Emily C; Moghadaszadeh, Behzad; Peacker, Bryan L; Leacock, Krystynne A; Horwitz, Naftali; Beggs, Alan H; Wagers, Amy J; Sabeti, Pardis C.
Affiliation
  • Tabebordbar M; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA. Electronic address: tabebord@fas.harvard.edu.
  • Lagerborg KA; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA; Harvard Program in Biological and Biomedical Sciences, Harvard Medical School, Boston, MA 02115, USA.
  • Stanton A; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA; Harvard Program in Virology, Harvard Medical School, Boston, MA 02115, USA.
  • King EM; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
  • Ye S; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA; Harvard-MIT Health Sciences and Technology, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
  • Tellez L; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
  • Krunnfusz A; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
  • Tavakoli S; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Harvard Stem Cell Institute, Cambridge, MA 02138, USA; Paul F. Glenn Center for the Biology of Aging, Harvard Medical School, Boston, MA 02115, USA; Stem Cell Program and Division of Hematology/Oncology,
  • Widrick JJ; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA.
  • Messemer KA; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Harvard Stem Cell Institute, Cambridge, MA 02138, USA; Paul F. Glenn Center for the Biology of Aging, Harvard Medical School, Boston, MA 02115, USA.
  • Troiano EC; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA.
  • Moghadaszadeh B; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA.
  • Peacker BL; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Harvard Stem Cell Institute, Cambridge, MA 02138, USA; Paul F. Glenn Center for the Biology of Aging, Harvard Medical School, Boston, MA 02115, USA.
  • Leacock KA; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Harvard Stem Cell Institute, Cambridge, MA 02138, USA; Paul F. Glenn Center for the Biology of Aging, Harvard Medical School, Boston, MA 02115, USA.
  • Horwitz N; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Harvard Stem Cell Institute, Cambridge, MA 02138, USA; Paul F. Glenn Center for the Biology of Aging, Harvard Medical School, Boston, MA 02115, USA; Section on Islet Cell and Regenerative Biology, Joslin
  • Beggs AH; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA.
  • Wagers AJ; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Harvard Stem Cell Institute, Cambridge, MA 02138, USA; Paul F. Glenn Center for the Biology of Aging, Harvard Medical School, Boston, MA 02115, USA; Section on Islet Cell and Regenerative Biology, Joslin
  • Sabeti PC; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA; Department of Organismic and Evolutionary Biology, FAS Center for Systems Biology, Harvard University, Cambridge, MA 02138, USA; Howard Hughes Medical Institute, Chevy Chase, MD 20815, USA. Electronic address: pardis@broadinstitute.org.
Cell ; 184(19): 4919-4938.e22, 2021 09 16.
Article in En | MEDLINE | ID: mdl-34506722
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Capsid / Gene Transfer Techniques / Dependovirus / Muscle, Skeletal / Directed Molecular Evolution Limits: Animals / Humans Language: En Journal: Cell Year: 2021 Type: Article
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Capsid / Gene Transfer Techniques / Dependovirus / Muscle, Skeletal / Directed Molecular Evolution Limits: Animals / Humans Language: En Journal: Cell Year: 2021 Type: Article