We invite you to discover Human Gene Therapy Clinical Development, a peer-reviewed journal publishing preclinical research and clinical trial data for gene and cell therapies.

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