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Molecular Therapy : the Journal of the American Society of Gene Therapy|January 20, 2007
Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNSGumei Liu, Yong Hong Chen, Xiaohua He, et al.
RNA (New York, N.Y.)|January 16, 2010
Structure and activity of putative intronic miRNA promotersAlex Mas Monteys, Ryan M Spengler, Ji Wan, et al.
Nature Medicine|September 23, 2003
Identification of PDGFR as a receptor for AAV-5 transductionGiovanni Di Pasquale, Beverly L Davidson, Colleen S Stein, et al.
Blood|May 12, 2005
Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transferYubin Kang, Litao Xie, Diane Thi Tran, et al.
Cell Reports|April 26, 2018
Cardiac mTORC1 Dysregulation Impacts Stress Adaptation and Survival in Huntington's DiseaseDaniel D Child, John H Lee, Christine J Pascua, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 26, 2009
Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease miceRyan L Boudreau, Jodi L McBride, Inês Martins, et al.
Human Molecular Genetics|September 25, 2009
Adenosine deamination in human transcripts generates novel microRNA binding sitesGlen M Borchert, Brian L Gilmore, Ryan M Spengler, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience|September 15, 2007
A knock-in reporter model of Batten diseaseSteven L Eliason, Colleen S Stein, Qinwen Mao, et al.
American Journal of Physiology. Lung Cellular and Molecular Physiology|April 30, 2013
Efficient delivery of RNA interference oligonucleotides to polarized airway epithelia in vitroShyam Ramachandran, Sateesh Krishnamurthy, Ashley M Jacobi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 28, 2011
Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's diseaseJodi L McBride, Mark R Pitzer, Ryan L Boudreau, et al.
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