Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning

By A. Aiuti, S. Slavin, M. Aker, Francesca Ficara, Sara Deola, A. Mortellaro, S. Morecki, G. Andolfi, A. Tabucchi, F. Carlucci and 7 more. Science, 2002

1,185 citations recorded by the source corpus.

Subfields: Genetics, Molecular Biology, Oncology

View Paper: https://www.semanticscholar.org/paper/6f5ec874103bf940cea8ecec9d4ba65055ca44fb

How researchers see this paper

The researchers whose trusted networks rank it highest, strongest advocate first. Every researcher gets a different order.

In Genetics

1. C. June
2. B. Levine
3. J. Kochenderfer
4. Jennifer N. Brudno
5. S. Grupp
6. Michel Sadelain

In Molecular Biology

1. Kenneth A Kim
2. M. Holmes
3. Frederic D. Bushman
4. Steven G. Deeks
5. C. June
6. A. Kinner
7. Rob Knight
8. D. Carroll

In Oncology

1. C. June
2. B. Levine
3. J. Kochenderfer
4. Jennifer N. Brudno
5. Michel Sadelain
6. Wendell A. Lim
7. S. Grupp

Citations in the corpus

• Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease (2010, background): “Characterization of a recurrent translocation t(2;3)(p15–22;q26) occurring in acute myeloid leukaemia.”
• Gene therapy clinical trials worldwide to 2012 – an update (2013, background): “In addition, the targeting of haematopoietic stem cells (HSCs) using an improved gene transfer protocol and a myeloablative conditioning regime underpin the more recent success [14,15].”
• Decade-Long Safety and Function of Retroviral-Modified Chimeric Antigen Receptor T-cells (2012, methodology): “To interrogate expression of CD4ζ, we isolated total RNA from 13 subjects 2–10 years post infusion, and measured transcriptional activity by RT-PCR for CD4ζ.”
• Zinc finger nuclease-mediated CCR5 knockout hematopoietic stem cell transplantation controls HIV-1 in vivo (2010, positive): “In addition, the experience of autologous HSC transplantation in gene therapy for patients with genetic diseases such as ADA-SCID (59–60), chronic granulomatous disease (61) and X-linked adrenoleukodystrophy (62), is that non-myeloablative conditioning can be used to facilitate engraftment of gene-m”