Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome
San Raffaele University of Rome · Bambino Gesù Children's Hospital · +15 more institutions
Abstract
Next-Generation Gene Therapy Few disciplines in contemporary clinical research have experienced the high expectations directed at the gene therapy field. However, gene therapy has been challenging to translate to the clinic, often because the therapeutic gene is expressed at insufficient levels in the patient or because the gene delivery vector integrates near protooncogenes, which can cause leukemia (see the Perspective by Verma ). Biffi et al. ( 1233158 , published online 11 July) and Aiuti et al. ( 1233151 ; published online 11 July) report progress on both fronts in gene therapy trials of three patients with metachromatic leukodystrophy (MLD), a neurodegenerative disorder, and three patients with…
Citation impact
- FWCI
- 89.38
- Percentile
- 100%
- References
- 62
Authors
40- AAAlessandro AiutiCorresponding
San Raffaele University of Rome, Bambino Gesù Children's Hospital, The San Raffaele Telethon Institute for Gene Therapy
- LBLuca Biasco
The San Raffaele Telethon Institute for Gene Therapy
- SSSamantha Scaramuzza
The San Raffaele Telethon Institute for Gene Therapy
- FFFrancesca Ferrua
Vita-Salute San Raffaele University, San Raffaele University of Rome, The San Raffaele Telethon Institute for Gene Therapy
- MPMaria Pia Cicalese
San Raffaele University of Rome, The San Raffaele Telethon Institute for Gene Therapy
Topics & keywords
- Genetic enhancement
- Wiskott–Aldrich syndrome
- Metachromatic leukodystrophy
- Stem cell
- Haematopoiesis
- Viral vector
- Medicine
- Hematopoietic stem cell
- Good health and well-being