Results 21 to 30 of about 1,182,904 (110)

Gene therapy Progress and Prospects: Gene therapy for the hemophilias [PDF]

Gene Therapy, 2003
Recent gene transfer trials for hemophilia A and B, bleeding disorders lacking either functional factor VIII or IX, respectively, have produced tantalizing results, suggesting that the potential to correct these bleeding disorders at a molecular level may be at hand.
openaire   +2 more sources

Gene therapy [PDF]

Nature, 2018
BIGGER BRIAN, GLEITZ HÉLÈNE
  +10 more sources

Gene therapy [PDF]

Current Biology, 1998
openaire   +2 more sources

Gene therapy [PDF]

Nature, 1992
openaire   +2 more sources

New Gene Therapy Strategy for β-Thalassemia. [PDF]

Stem Cell Rev Rep
Liang D, Schmidt-Wolf IGH, Pu J.
europepmc   +1 more source

Gene Therapy [PDF]

BMJ, 1989
openaire   +2 more sources

Activity-dependent antioxidant gene therapy protects against epileptogenesis. [PDF]

Signal Transduct Target Ther
Singh PK, Lignani G, Walker MC, Lieb A, Shekh-Ahmad T.   +4 more
europepmc   +1 more source

Gene Therapy for Heart Failure. [PDF]

J Cardiovasc Transl Res
Wang H, Määttä A, Ylä-Herttuala S.
europepmc   +1 more source

Progress and challenges in intrathecal gene therapy for neurological disorders. [PDF]

EBioMedicine
Kagiava A, Chen X, Finkel RS, Gray SJ, Kleopa KA.   +4 more
europepmc   +1 more source

Hematopoietic Stem-Cell Gene Therapy for Cystinosis. [PDF]

N Engl J Med
Barshop BA, Ball ED, Benador N, Trauner D, Phillips S, Dohil R, Afshari NA, Roy S, Campo Fernandes B, Kohn D, Shayan K, Everett JK, Bushman FD, Midgley J, Liang H, Sawyers A, Gangoiti JA, Panchal M, Ahmed I, Cherqui S.   +19 more
europepmc   +1 more source