Long-Term Follow-Up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy-Reference-Cited by-同舟云学术

Long-Term Follow-Up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

Published:2021-10-01 Issue:19-20 Volume:32 Page:1260-1269
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Bougnères Pierre¹²³^ORCID,Hacein-Bey-Abina Salima⁴⁵,Labik Ivan⁶,Adamsbaum Catherine⁷,Castaignède Clémence⁸^ORCID,Bellesme Céline⁸^ORCID,Schmidt Manfred⁶

Affiliation:

1. UMR1195 INSERM, Le Kremlin Bicêtre, France

2. Université Paris Saclay, MIRCen Institute/Neuratris, Fontenay-aux-Roses, France

3. Therapy Design Consulting, Vincennes, France

4. Clinical Immunology Laboratory, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris Saclay, Paris, France

5. UTCBS, CNRS UMR8258, INSERM U1267, Faculté de Pharmacie de Paris, Université de Paris, Le Kremlin-Bicêtre, France;

6. GeneWerk GmbH, Heidelberg, Germany

7. Pediatric Radiology and

8. Pediatric Neurology, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris Saclay, Le Kremlin-Bicêtre, France.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2021.053

Reference36 articles.

1. Putative X-linked adrenoleukodystrophy gene shares unexpected homology with ABC transporters

2. X-linked adrenoleukodystrophy (X-ALD): clinical presentation and guidelines for diagnosis, follow-up and management

3. The Changing Face of Adrenoleukodystrophy

4. Hematopoietic cells differentiate into both microglia and macroglia in the brains of adult mice

5. Targeting gene-modified hematopoietic cells to the central nervous system: Use of green fluorescent protein uncovers microglial engraftment

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