Abstract

Administrative Core Dr. James M. Wilson will serve as Director of the Administrative Core in his capacity as PI of the P01. Drs. Daniel J. Rader and Arthur Caplan are Associate Directors of this Core. The administrative home of the grant is the Gene Therapy Program (GTP) at Penn that is directed by Dr. Wilson. Administrative staff at GTP report to Dr. Wilson and will have complete responsibility for the administrative management of the P01. A number of mechanisms are in place to manage the science and interactions between the groups including an FH Team that is staffed with an experienced program manager and meets frequently. Several important internal and external committees provide oversight and advice including: Internal ? Shared Resource Committee of the School of Medicine that reviews the Vector Core annually, Management Committee of GTP that meets weekly; External - an External Scientific Advisory Committee and Ethics Advisory Board who meet with leadership of the P01 once per year.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Research Program Projects (P01)
Project #
2P01HL059407-16A1
Application #
8935364
Study Section
Heart, Lung, and Blood Initial Review Group (HLBP)
Project Start
2000-05-15
Project End
2020-05-31
Budget Start
2015-08-01
Budget End
2016-05-31
Support Year
16
Fiscal Year
2015
Total Cost
$141,734
Indirect Cost
$53,150

  Institution

Name
University of Pennsylvania
Department
Type
DUNS #
042250712
City
Philadelphia
State
PA
Country
United States
Zip Code
19104

  Publications

Giles, April R; Govindasamy, Lakshmanan; Somanathan, Suryanarayan et al. (2018) Mapping an Adeno-associated Virus 9-Specific Neutralizing Epitope To Develop Next-Generation Gene Delivery Vectors. J Virol 92:
Amengual, Jaume; Guo, Liang; Strong, Alanna et al. (2018) Autophagy Is Required for Sortilin-Mediated Degradation of Apolipoprotein B100. Circ Res 122:568-582
Giles, April R; Sims, Joshua J; Turner, Kevin B et al. (2018) Deamidation of Amino Acids on the Surface of Adeno-Associated Virus Capsids Leads to Charge Heterogeneity and Altered Vector Function. Mol Ther 26:2848-2862
Calcedo, Roberto; Somanathan, Suryanarayan; Qin, Qiuyue et al. (2017) Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for ?-1-antitrypsin deficiency. Proc Natl Acad Sci U S A 114:1655-1659
Ai, Jianzhong; Li, Jia; Gessler, Dominic J et al. (2017) Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery. Sci Rep 7:40336
Ai, Jianzhong; Tai, Phillip W L; Lu, Yi et al. (2017) Characterization of adenoviral transduction profile in prostate cancer cells and normal prostate tissue. Prostate 77:1265-1270
Greig, Jenny A; Limberis, Maria P; Bell, Peter et al. (2017) Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR+/- Rhesus Macaques. Hum Gene Ther Clin Dev 28:39-50
Greig, Jenny A; Limberis, Maria P; Bell, Peter et al. (2017) Nonclinical Pharmacology/Toxicology Study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a Mouse Model of Homozygous Familial Hypercholesterolemia. Hum Gene Ther Clin Dev 28:28-38
Rashnonejad, Afrooz; Chermahini, Gholamhossein Amini; Li, Shaoyong et al. (2016) Large-Scale Production of Adeno-Associated Viral Vector Serotype-9 Carrying the Human Survival Motor Neuron Gene. Mol Biotechnol 58:30-6
Ajufo, Ezim; Cuchel, Marina (2016) Recent Developments in Gene Therapy for Homozygous Familial Hypercholesterolemia. Curr Atheroscler Rep 18:22

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