Publication 11/01/2016

Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice.

Hinderer C, Katz N, Louboutin JP, Bell P, Yu H, Nayal M, Kozarsky K, O'Brien WT, Goode T, Wilson JM

Human Gene Therapy

Publication 11/01/1995

A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia.

Grossman M, Rader DJ, Muller DW, Kolansky DM, Kozarsky K, Clark BJ 3rd, Stein EA, Lupien PJ, Brewer HB Jr, Raper SE

Nature Medicine

Publication 4/01/1994

Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia.

Grossman M, Raper SE, Kozarsky K, Stein EA, Engelhardt JF, Muller D, Lupien PJ, Wilson JM

Nature Genetics

Publication 1/01/1994

Efficient catheter-mediated gene transfer into the heart using replication-defective adenovirus.

Barr E, Carroll J, Kalynych AM, Tripathy SK, Kozarsky K, Wilson JM, Leiden JM

Gene Therapy

Publication 9/01/1993

Adenovirus-mediated correction of the genetic defect in hepatocytes from patients with familial hypercholesterolemia.

Kozarsky K, Grossman M, Wilson JM

Somatic Cell And Molecular Genetics

Publication 5/01/1993

Direct gene transfer of human CFTR into human bronchial epithelia of xenografts with E1-deleted adenoviruses.

Engelhardt JF, Yang Y, Stratford-Perricaudet LD, Allen ED, Kozarsky K, Perricaudet M, Yankaskas JR, Wilson JM

Nature Genetics