The administrative core of this translational program is integral to the Horae Gene Therapy Center (GTC) of UMMS, and provides the mechanism for pre- and post-award administration of the program, for convening of the investigators who constitute the key personnel of the subprojects and cores, and for assembling the External Advisory Committee to provide ongoing peer review and input to all programmatic initiatives. The administrative core interacts at all levels with the central administration of UMMS (including the office of the Provost/Dean, the Office of Research, and the Center for Clinical and Translational Science). Finally, the Administrative core will provide assistance to the communication of results through peer review publications and presentations at national meetings relevant to pulmonary disease, gene therapy, and alpha-1 antitrypsin deficiency, specifically.

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Research Program Projects (P01)
Project #
5P01HL131471-04
Application #
9695018
Study Section
Special Emphasis Panel (ZHL1)
Program Officer
Postow, Lisa
Project Start
Project End
Budget Start
2019-05-01
Budget End
2020-04-30
Support Year
4
Fiscal Year
2019
Total Cost
Indirect Cost
Name
University of Massachusetts Medical School Worcester
Department
Type
DUNS #
603847393
City
Worcester
State
MA
Country
United States
Zip Code
01655
Song, Chun-Qing; Xue, Wen (2018) CRISPR-Cas-related technologies in basic and translational liver research. Nat Rev Gastroenterol Hepatol 15:251-252
Zhang, Wei; Li, Linjing; Su, Qin et al. (2018) Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis. Hum Gene Ther 29:42-50
Tai, Phillip W L; Xie, Jun; Fong, Kaiyuen et al. (2018) Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras. Mol Ther Methods Clin Dev 9:130-141
Borel, Florie; Sun, Huaming; Zieger, Marina et al. (2018) Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema. Proc Natl Acad Sci U S A 115:2788-2793
Zhang, Xiao-Ou; Fu, Yu; Mou, Haiwei et al. (2018) The temporal landscape of recursive splicing during Pol II transcription elongation in human cells. PLoS Genet 14:e1007579
Wang, Dan; Gao, Guangping (2018) Taking a Hint from Structural Biology: To Better Understand AAV Transport across the BBB. Mol Ther 26:336-338
Wang, Dan; Li, Jia; Tran, Karen et al. (2018) Slow Infusion of Recombinant Adeno-Associated Viruses into the Mouse Cerebrospinal Fluid Space. Hum Gene Ther Methods 29:75-85
Yin, Hao; Song, Chun-Qing; Suresh, Sneha et al. (2018) Partial DNA-guided Cas9 enables genome editing with reduced off-target activity. Nat Chem Biol 14:311-316
Keeler, Allison M; Zieger, Marina; Todeasa, Sophia H et al. (2018) Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease. Hum Gene Ther :
Wang, Dan; Li, Shaoyong; Gessler, Dominic J et al. (2018) A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates. Mol Ther Methods Clin Dev 9:234-246

Showing the most recent 10 out of 42 publications