Publication
2/01/2004
AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.
Ziegler RJ, Lonning SM, Armentano D, Li C, Souza DW, Cherry M, Ford C, Barbon CM, Desnick RJ, Gao G, Wilson JM, Peluso R, Godwin S, Carter BJ, Gregory RJ, Wadsworth SC, Cheng SH
Molecular Therapy : The Journal Of The American Society Of Gene Therapy
Publication
9/01/1992
Human cystic fibrosis transmembrane conductance regulator directed to respiratory epithelial cells of transgenic mice.
Whitsett JA, Dey CR, Stripp BR, Wikenheiser KA, Clark JC, Wert SE, Gregory RJ, Smith AE, Cohn JA, Wilson JM
Nature Genetics
