RT Book, Section
A1 Fung, Hua
A1 Gerson, Stanton
A2 Kaushansky, Kenneth
A2 Lichtman, Marshall A.
A2 Prchal, Josef T.
A2 Levi, Marcel M.
A2 Press, Oliver W.
A2 Burns, Linda J.
A2 Caligiuri, Michael
SR Print(0)
ID 1121091086
T1 Gene Therapy for Hematologic Diseases
T2 Williams Hematology, 9e
YR 2015
FD 2015
PB McGraw-Hill Education
PP New York, NY
SN 9780071833004
LK hemonc.mhmedical.com/content.aspx?aid=1121091086
RD 2024/04/19
AB SUMMARYThe  term  gene  therapy  describes  treatment  resulting  from  expression  of  a  transferred  gene  (or  transgene)  in  diseased  or  other  cells  by  engineered  vectors.  Once  within  the  cell,  the  transgene  can  direct  synthesis  of  a  therapeutic  protein  that  can  complement  a  genetic  deficiency  or  confer  upon  the  cell  a  desired  phenotype  or  function.  Many  clinical  trials  have  involved  gene  therapy  for  patients  with  various  gene-deficient  hematologic  diseases,  such  as  severe  combined  immunodeficiency,  hemophilia,  Wiskott-Aldrich  syndrome,  chronic  granulomatous  disease,  aplastic  anemia,  hemoglobinopathies,  HIV  infection,  and  leukemia.  Results  from  some  clinical  trials  indicate  that  gene  therapy  can  cure  or  improve  many  inherited  or  acquired  hematologic  disorders.  This  chapter  reviews  the  basic  principles  of  gene  transfer  and  the  results  of  selected  preclinical  and  clinical  studies.