Publication
6/07/2017
5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.
Mueller C, Gernoux G, Gruntman AM, Borel F, Reeves EP, Calcedo R, Rouhani FN, Yachnis A, Humphries M, Campbell-Thompson M, Messina L, Chulay JD, Trapnell B, Wilson JM, McElvaney NG, Flotte TR
Molecular Therapy : The Journal Of The American Society Of Gene Therapy
Publication
5/01/2017
Characterization of Adeno-Associated Viral Vector-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice.
Greig JA, Wang Q, Reicherter AL, Chen SJ, Hanlon AL, Tipper CH, Clark KR, Wadsworth S, Wang L, Wilson JM
Human Gene Therapy
Publication
4/01/2017
Chronic Hyperphosphatemia and Vascular Calcification Are Reduced by Stable Delivery of Soluble Klotho.
Hum JM, O'Bryan LM, Tatiparthi AK, Cass TA, Clinkenbeard EL, Cramer MS, Bhaskaran M, Johnson RL, Wilson JM, Smith RC, White KE
Journal Of The American Society Of Nephrology : Jasn
Publication
4/01/2017
AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice.
Wang L, Bell P, Morizono H, He Z, Pumbo E, Yu H, White J, Batshaw ML, Wilson JM
Molecular Genetics And Metabolism
Publication
4/01/2017
Regulatory and Exhausted T Cell Responses to AAV Capsid.
Gernoux G, Wilson JM, Mueller C
Human Gene Therapy
Publication
3/01/2017
Nonclinical Pharmacology/Toxicology Study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a Mouse Model of Homozygous Familial Hypercholesterolemia.
Greig JA, Limberis MP, Bell P, Chen SJ, Calcedo R, Rader DJ, Wilson JM
Human Gene Therapy. Clinical Development