L
Lili Wang
Researcher at University of Pennsylvania
Publications - 103
Citations - 9261
Lili Wang is an academic researcher from University of Pennsylvania. The author has contributed to research in topics: Genetic enhancement & Adeno-associated virus. The author has an hindex of 41, co-authored 98 publications receiving 8319 citations. Previous affiliations of Lili Wang include Fudan University & University of North Carolina at Chapel Hill.
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Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
TL;DR: Vectors based on AAV7 and AAV8 should be considered for human gene therapy because of low reactivity to antibodies directed to human AAVs and because gene transfer efficiency in muscle was similar to that obtained with the best known serotype, whereas, in liver, gene transfer was substantially higher than previously described.
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Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial
William W. Hauswirth,Tomas S. Aleman,Shalesh Kaushal,Artur V. Cideciyan,Sharon B. Schwartz,Lili Wang,Thomas J. Conlon,Sanford L. Boye,Terence R. Flotte,Barry J. Byrne,Samuel G. Jacobson +10 more
TL;DR: A recombinant adeno-associated virus serotype 2 vector, altered to carry the human RPE65 gene (rAAV2-CBSB-hRPE65) restored vision in animal models with R PE65 deficiency, and Comparisons are drawn between the present work and two other studies of ocular gene therapy for RPE 65-LCA that were carried out contemporaneously and reported.
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A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice
Yang Yang,Lili Wang,Peter Bell,Deirdre McMenamin,Zhenning He,John H. White,Hongwei Yu,Chenyu Xu,Hiroki Morizono,Kiran Musunuru,Mark L. Batshaw,James M. Wilson +11 more
TL;DR: In this paper, a CRISPR-Cas9-based approach was used for gene correction in newborn mice with a partial deficiency in the urea cycle disorder enzyme, ornithine transcarbamylase (OTC).
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Chronic suppression of heart-failure progression by a pseudophosphorylated mutant of phospholamban via in vivo cardiac rAAV gene delivery
Masahiko Hoshijima,Yasuhiro Ikeda,Yoshitaka Iwanaga,Susumu Minamisawa,Moto-o Date,Yusu Gu,Mitsuo Iwatate,Manxiang Li,Lili Wang,James M. Wilson,Yibin Wang,John Ross,Kenneth R. Chien +12 more
TL;DR: Transcoronary gene transfer of S16EPLN via rAAV vector is a potential therapy for progressive dilated cardiomyopathy and associated heart failure and protecting cardiac myocytes from cytopathic plasma-membrane disruption.
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Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
Mark L. Brantly,Jeffrey D. Chulay,Lili Wang,Christian Mueller,Margaret Humphries,L. Terry Spencer,Farshid N. Rouhani,Thomas J. Conlon,Roberto Calcedo,Michael R. Betts,Carolyn T. Spencer,Barry J. Byrne,James M. Wilson,Terence R. Flotte +13 more
TL;DR: It is suggested that immune responses to AAV capsid that develop after i.m. injection of a serotype 1 rAAV vector expressing AAT do not completely eliminate transduced cells in this context.