L
Leah C. Byrne
Researcher at University of Pittsburgh
Publications - 46
Citations - 1885
Leah C. Byrne is an academic researcher from University of Pittsburgh. The author has contributed to research in topics: Gene delivery & Retinal. The author has an hindex of 12, co-authored 41 publications receiving 1437 citations. Previous affiliations of Leah C. Byrne include Helen Wills Neuroscience Institute & University of Pennsylvania.
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Journal ArticleDOI
In Vivo???Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous
Deniz Dalkara,Leah C. Byrne,Ryan R. Klimczak,Meike Visel,Lu Yin,William H. Merigan,John G. Flannery,John G. Flannery,David V. Schaffer,David V. Schaffer +9 more
TL;DR: It is shown that delivery of a new vector into the eye’s easily accessible vitreous humour transduces the entire retina and rescues degenerative eye disease phenotypes, suggesting that gene therapy vectors can be designed to penetrate dense tissues, which currently constitute barriers to gene delivery.
Journal ArticleDOI
Rod-derived cone viability factor promotes cone survival by stimulating aerobic glycolysis.
Najate Aït-Ali,Najate Aït-Ali,Najate Aït-Ali,Ram Fridlich,Ram Fridlich,Ram Fridlich,Géraldine Millet-Puel,Géraldine Millet-Puel,Géraldine Millet-Puel,Emmanuelle Clérin,Emmanuelle Clérin,Emmanuelle Clérin,François Delalande,François Delalande,Céline Jaillard,Céline Jaillard,Céline Jaillard,Frédéric Blond,Frédéric Blond,Frédéric Blond,Ludivine Perrocheau,Ludivine Perrocheau,Ludivine Perrocheau,Sacha Reichman,Sacha Reichman,Sacha Reichman,Leah C. Byrne,Anne Olivier-Bandini,Jacques Bellalou,Emmanuel Moyse,Frédéric Bouillaud,Frédéric Bouillaud,Frédéric Bouillaud,Xavier Nicol,Xavier Nicol,Xavier Nicol,Deniz Dalkara,Deniz Dalkara,Deniz Dalkara,Alain Van Dorsselaer,Alain Van Dorsselaer,José-Alain Sahel,José-Alain Sahel,José-Alain Sahel,Thierry Léveillard,Thierry Léveillard,Thierry Léveillard +46 more
TL;DR: It is shown that RdCVF acts through binding to Basigin-1 (BSG1), a transmembrane protein expressed specifically by photoreceptors, resulting in increased glucose entry into cones, and an entirely novel mechanism of neuroprotection through the stimulation of glucose metabolism.
Journal ArticleDOI
Viral-mediated RdCVF and RdCVFL expression protects cone and rod photoreceptors in retinal degeneration
Leah C. Byrne,Deniz Dalkara,Gabriel Luna,Steven K. Fisher,Emmanuelle Clérin,José-Alain Sahel,Thierry Léveillard,John G. Flannery +7 more
TL;DR: Results indicate different functions for Nxnl1 isoforms in the retina and suggest that RdCVF gene therapy has potential for treating retinal degenerative disease.
Journal ArticleDOI
In vivo genome editing improves motor function and extends survival in a mouse model of ALS
Thomas Gaj,David S. Ojala,Freja K. Ekman,Leah C. Byrne,Prajit Limsirichai,David V. Schaffer,David V. Schaffer +6 more
TL;DR: The potential for CRISPR-Cas9 to treat SOD1-linked forms of ALS and other central nervous system disorders caused by autosomal dominant mutations is illustrated.
Journal ArticleDOI
Massively parallel cis-regulatory analysis in the mammalian central nervous system
Susan Q. Shen,Connie A. Myers,Andrew E. O. Hughes,Leah C. Byrne,John G. Flannery,Joseph C. Corbo +5 more
TL;DR: This work adapts exome capture technology to instead capture candidate CREs, thereby tiling across the targeted regions and markedly increasing the length of CREs that can be readily assayed, and packages the library into adeno-associated virus (AAV), thereby allowing delivery to target organs in vivo.