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

Adrenoleukodystrophy / diagnostic imaging Follow-Up Studies Genetic Therapy Hematopoietic Stem Cell Transplantation Hematopoietic Stem Cells Humans Male
cerebral adrenoleukodystrophy gene therapy hematopoietic stem cells lentivirus

Journal

Human gene therapy
ISSN: 1557-7422
Titre abrégé: Hum Gene Ther
Pays: United States
ID NLM: 9008950

Informations de publication

Date de publication:
10 2021
Historique:
pubmed: 2 4 2021
medline: 1 2 2022
entrez: 1 4 2021
Statut: ppublish

Résumé

In 2009, cerebral adrenoleukodystrophy (c-ALD) became the first brain disease to be treated with lentiviral (LV)-based hematopoietic stem cell gene therapy with the

Identifiants

DOI: 10.1089/hum.2021.053 PMID: 33789438
pubmed: 33789438
doi: 10.1089/hum.2021.053
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1260-1269

Auteurs

Pierre Bougnères (P)

UMR1195 INSERM, Le Kremlin Bicêtre, France.
Université Paris Saclay, MIRCen Institute/Neuratris, Fontenay-aux-Roses, France.
Therapy Design Consulting, Vincennes, France.
ORCID: 0000-0003-0017-4572

Salima Hacein-Bey-Abina (S)

Clinical Immunology Laboratory, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris Saclay, Paris, France.
UTCBS, CNRS UMR8258, INSERM U1267, Faculté de Pharmacie de Paris, Université de Paris, Le Kremlin-Bicêtre, France.

Ivan Labik (I)

GeneWerk GmbH, Heidelberg, Germany.

Catherine Adamsbaum (C)

Pediatric Radiology and.

Clémence Castaignède (C)

Pediatric Neurology, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris Saclay, Le Kremlin-Bicêtre, France.
ORCID: 0000-0002-7888-4286

Céline Bellesme (C)

Pediatric Neurology, Hôpital Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, Université Paris Saclay, Le Kremlin-Bicêtre, France.
ORCID: 0000-0002-5733-711X

Manfred Schmidt (M)

GeneWerk GmbH, Heidelberg, Germany.

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Classifications MeSH

questionsmedicales.fr › Maladies endocriniennes › Maladies des surrénales › Insuffisance surrénale › Adrénoleucodystrophie › Long-Term Follow-Up of Hematopoietic Stem-Cell...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études épidémiologiques › Études de cohortes › Études de suivi › Long-Term Follow-Up of Hematopoietic Stem-Cell...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Long-Term Follow-Up of Hematopoietic Stem-Cell...
questionsmedicales.fr › Procédures de chirurgie opératoire › Transplantation › Transplantation cellulaire › Transplantation de cellules souches › Transplantation de cellules souches hématopoïétiques › Long-Term Follow-Up of Hematopoietic Stem-Cell...
questionsmedicales.fr › Systèmes sanguin et immunitaire › Système hématopoïétique › Cellules de la moelle osseuse › Cellules souches hématopoïétiques › Long-Term Follow-Up of Hematopoietic Stem-Cell...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Long-Term Follow-Up of Hematopoietic Stem-Cell...