Gene replacement therapy in a schwannoma mouse model of neurofibromatosis type 2.

Schwann cells adeno-associated viral vector gene therapy neurofibromatosis type 2 schwannoma

Journal

Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857

Informations de publication

Date de publication:
08 Sep 2022
Historique:
received: 14 01 2022
accepted: 17 06 2022
entrez: 18 7 2022
pubmed: 19 7 2022
medline: 19 7 2022
Statut: epublish

Résumé

Loss of function of the neurofibromatosis type 2 (NF2) tumor suppressor gene leads to the formation of schwannomas, meningiomas, and ependymomas, comprising ∼50% of all sporadic cases of primary nervous system tumors. NF2 syndrome is an autosomal dominant condition, with bi-allelic inactivation of germline and somatic alleles resulting in loss of function of the encoded protein merlin and activation of mammalian target of rapamycin (mTOR) pathway signaling in

Identifiants

DOI: 10.1016/j.omtm.2022.06.012 PMID: 35846573 PMC: PMC9263409
pubmed: 35846573
doi: 10.1016/j.omtm.2022.06.012
pii: S2329-0501(22)00091-2
pmc: PMC9263409
doi:

Types de publication

Journal Article

Langues

eng

Pagination

169-180

Subventions

Organisme : NINDS NIH HHS
ID : R01 NS113854
Pays : United States

Informations de copyright

© 2022 The Author(s).

Déclaration de conflit d'intérêts

C.A.M. has a financial interest in Chameleon Biosciences, Inc. and Sphere Gene Therapeutics, companies developing an enveloped AAV vector platform technology. C.A.M. also has a financial interest in Skylark Bio, which is developing gene therapy to treat hereditary hearing loss. C.A.M.’s interests were reviewed and are managed by MGH and Partners HealthCare in accordance with their conflict of interest policies. D.B.W. is a consultant for Skylark Bio, Inc., Recursion Pharmaceuticals, Mulberry Biotherapeutics, and NF2 Biosolutions. The other authors declare no competing interests.

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Auteurs

Shilpa Prabhakar (S)

Department of Neurology and Center for Molecular Imaging Research, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA.

Roberta L Beauchamp (RL)

Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.

Pike See Cheah (PS)

Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Center for Molecular Imaging Research, Massachusetts General Hospital, 25 Shattuck St, Boston, MA 02115, USA.
Department of Human Anatomy, Faculty of Medicine and Health Sciences, Universiti Putra Malaysia, JALAN UNIVERSITI 1 Serdang, 43400 Seri Kembangan, Selangor, Malaysia.

Akiko Yoshinaga (A)

Department of Neurology and Center for Molecular Imaging Research, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02129, USA.

Edwina Abou Haidar (EA)

Department of Neurology and Center for Molecular Imaging Research, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA.

Sevda Lule (S)

Department of Neurology and Center for Molecular Imaging Research, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02129, USA.

Gayathri Mani (G)

Department of Neurology and Center for Molecular Imaging Research, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02129, USA.

Katia Maalouf (K)

Department of Neurology and Center for Molecular Imaging Research, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA.

Anat Stemmer-Rachamimov (A)

Department of Pathology and Center for Cancer Research, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02114, USA.

David H Jung (DH)

Department of Otolaryngology, Massachusetts Eye and Ear and Harvard Medical School, Boston, MA 02114, USA.
Program in Speech and Hearing Bioscience and Technology, Harvard Medical School, Boston, MA 02114, USA.

D Bradley Welling (DB)

Department of Otolaryngology Head and Neck Surgery, Harvard Medical School, Massachusetts Eye and Ear and Massachusetts General Hospital, Boston, MA 02114, USA.
Program in Speech and Hearing Bioscience and Technology, Harvard Medical School, Boston, MA 02114, USA.

Marco Giovannini (M)

Department of Head and Neck Surgery, David Geffen School of Medicine at UCLA and Jonsson Comprehensive Cancer Center (JCCC), University of California Los Angeles, Los Angeles, CA 90095, USA.

Scott R Plotkin (SR)

Department of Neurology and Cancer Center, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02114, USA.

Casey A Maguire (CA)

Department of Neurology, Massachusetts General Hospital and NeuroDiscovery Center, Harvard Medical School, Boston, MA 02114, USA.

Vijaya Ramesh (V)

Department of Neurology and Center for Genomic Medicine, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02114, USA.

Xandra O Breakefield (XO)

Department of Neurology and Center for Molecular Imaging Research, Department of Radiology, Massachusetts General Hospital and Harvard Medical School, Boston, MA 02114, USA.

Classifications MeSH