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Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal.
Moscatelli, Ilana; Almarza, Elena; Schambach, Axel; Ricks, David; Schulz, Ansgar; Herzog, Christopher D; Henriksen, Kim; Askmyr, Maria; Schwartz, Jonathan D; Richter, Johan.
Afiliação
  • Moscatelli I; Department of Molecular Medicine and Gene Therapy, Lund Strategic Center for Stem Cell Biology, Lund University, Lund, Sweden.
  • Almarza E; Rocket Pharmaceuticals, Inc., New York, NY, USA.
  • Schambach A; Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
  • Ricks D; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
  • Schulz A; Rocket Pharmaceuticals, Inc., New York, NY, USA.
  • Herzog CD; Department of Pediatrics and Adolescent Medicine, University Medical Center Ulm, Germany.
  • Henriksen K; Rocket Pharmaceuticals, Inc., New York, NY, USA.
  • Askmyr M; Nordic Bioscience A/S, Herlev, Denmark.
  • Schwartz JD; Department of Molecular Medicine and Gene Therapy, Lund Strategic Center for Stem Cell Biology, Lund University, Lund, Sweden.
  • Richter J; Rocket Pharmaceuticals, Inc., New York, NY, USA.
Mol Ther Methods Clin Dev ; 20: 389-397, 2021 Mar 12.
Article em En | MEDLINE | ID: mdl-33575431
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Texto completo: 1 Base de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Suécia
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Suécia