Lentiviral Hematopoietic Stem Cell Gene Therapy in Inherited Metabolic Disorders

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Wagemaker G.

HUMAN GENE THERAPY, cilt.25, sa.10, ss.862-865, 2014 (SCI-Expanded) identifier identifier identifier

  • Yayın Türü: Makale / Derleme
  • Cilt numarası: 25 Sayı: 10
  • Basım Tarihi: 2014
  • Doi Numarası: 10.1089/hum.2014.102
  • Dergi Adı: HUMAN GENE THERAPY
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus
  • Sayfa Sayıları: ss.862-865
  • Hacettepe Üniversitesi Adresli: Hayır

Özet

After more than 20 years of development, lentiviral hematopoietic stem cell gene therapy has entered the stage of initial clinical implementation for immune deficiencies and storage disorders. This brief review summarizes the development and applications, focusing on the lysosomal enzyme deficiencies, especially Pompe disease.