Gene Therapy for Genetic Disease

被引：0
|
作者：
邱信芳
薛京伦
机构：
[1] Fudan University
[2] PRC
[3] Shanghai 200433
[4] State Key Laboratory of Genetic Engineering
来源：
ProgressinNaturalScience | 1994年 / 05期
关键词：
clotting factor IX; gene therapy; gene transfer; hemophilia B;
D O I：
暂无
中图分类号：
R596 [遗传性疾病];
学科分类号：
1002 ; 100201 ;
摘要：
There are more than 4700 kinds of genetic diseases and millions of sufferers worldwide. The deficient genes not only cause suffering to the patients, but bring tragedy to their descendants through heredity.People have been trying to use gene therapy strategy to cure hereditary diseases. With the development of technology, the laboratorial somatic cell gene therapy is moving towards clinical bed and has shown promising prospect. Hemophilia B is one of the several kinds of diseases that are suitable for somatic cell gene therapy owing to its small pathogenic gene and clear genetic
引用
收藏
页码：61 / 69
页数：9
相关论文
共 50 条
  • [21] Fetal somatic gene therapy - A preventive approach to the treatment of genetic disease: The case for
    Coutelle, C
    Themis, M
    Schneider, H
    Kiserud, I
    Cook, T
    Douar, AM
    Hanson, M
    Pavirani, A
    Rodeck, C
    STEM CELLS FORM CORD BLOOD, IN UTERO STEM CELL DEVELOPMENT, AND TRANSPLANTATION-INCLUSIVE GENE THERAPY, 2001, 33 : 99 - 114
  • [22] Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease
    Hoban, Megan D.
    Orkin, Stuart H.
    Bauer, Daniel E.
    BLOOD, 2016, 127 (07) : 839 - 848
  • [23] Preemptive therapy for genetic disease
    Blau, CA
    Stamatoyannopoulos, G
    NATURE MEDICINE, 1996, 2 (02) : 161 - 162
  • [24] Gene therapy for genetic muscle diseases
    Karpati, G.
    NEUROMUSCULAR DISORDERS, 2006, 16 : S191 - S191
  • [25] A REVIEW ON GENE THERAPY FOR THE GENETIC REVOLUTION
    Shivappa, Nagoba N.
    Sonali, Khurde S.
    Atiya, Shaikh L.
    Krishna, Shimge R.
    INDO AMERICAN JOURNAL OF PHARMACEUTICAL SCIENCES, 2018, 5 (07): : 7111 - 7122
  • [26] Gene Therapy for Rare Genetic Diseases
    Lundstrom, Kenneth
    Yannaki, Evangelia
    Chou, Janice
    Saad, Fawzy A.
    CURRENT GENE THERAPY, 2024, 24 (01) : 4 - 5
  • [27] GENE THERAPY AND ETHICS OF GENETIC THERAPEUTICS
    NEVILLE, R
    ANNALS OF THE NEW YORK ACADEMY OF SCIENCES, 1976, 265 (JAN23) : 153 - 161
  • [28] Gene therapy strategies for genetic leukodystrophies
    Cartier, N.
    Sevin, C.
    Hacein-Bey-Abina, S.
    Piguet, F.
    Bartholomae, C.
    Colle, M. A.
    Schmitt, M.
    Fischer, A.
    Cavazzana-Calvo, M.
    Cherel, Y.
    Raoul, S.
    Roujeau, T.
    Zerah, M.
    Crystal, R.
    Von Kalle, C.
    Aubourg, P.
    FEBS JOURNAL, 2011, 278 : 68 - 68
  • [29] Gene Therapy for Acquired and Genetic Cholestasis
    Martinez-Garcia, Javier
    Molina, Angie
    Gonzalez-Aseguinolaza, Gloria
    Weber, Nicholas D.
    Smerdou, Cristian
    BIOMEDICINES, 2022, 10 (06)
  • [30] GENETIC THERAPY - SOMATIC GENE TRANSPLANTS
    CASKEY, CT
    HOSPITAL PRACTICE, 1987, 22 (08): : 181 - &
12345