Search Results - "Pyne NK"

Comparative analysis of cell-specific promoters in AAV9-mediated gene therapy targeting the central nervous system.

Authors: Chornyy S Herstine JA Holaway C et al.

Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2025 Sep 05; Vol. 33 (4), pp. 101588. Date of Electronic Publication: 2025 Sep 05 (Print Publication: 2025).

Publication Type: Journal Article

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE

Age-sensitive response of systemic AAV-mediated gene therapy in a newly characterized feline model of mucolipidosis II.

Authors: Pyne NK Bagel J Shyng C et al.

Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Aug 06; Vol. 33 (8), pp. 3808-3821. Date of Electronic Publication: 2025 Apr 24.

Publication Type: Journal Article

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE

Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease.

Authors: Herstine JA Chang PK Chornyy S et al.

Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Jun 05; Vol. 32 (6), pp. 1701-1720. Date of Electronic Publication: 2024 Mar 27.

Publication Type: Journal Article; Research Support, Non-U.S. Gov't; Research Support, N.I.H., Extramural

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE

Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models.

Authors: Morelli KH Griffin LB Pyne NK et al.

Source: The Journal of clinical investigation [J Clin Invest] 2019 Dec 02; Vol. 129 (12), pp. 5568-5583.

Publication Type: Journal Article; Research Support, N.I.H., Extramural

Journal Info: Publisher: American Society for Clinical Investigation Country of Publication: United States NLM ID: 7802877 Publication Model: Print Cited Medium: Internet ISSN: 1558-8238 (Electronic) Linking ISSN: 00219738 NLM ISO Abbreviation: J Clin Invest Subsets: MEDLINE

Aberrant splicing in transgenes containing introns, exons, and V5 epitopes: lessons from developing an FSHD mouse model expressing a D4Z4 repeat with flanking genomic sequences.

Authors: Ansseau E Domire JS Wallace LM et al.

Source: PloS one [PLoS One] 2015 Mar 05; Vol. 10 (3), pp. e0118813. Date of Electronic Publication: 2015 Mar 05 (Print Publication: 2015).

Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

Journal Info: Publisher: Public Library of Science Country of Publication: United States NLM ID: 101285081 Publication Model: eCollection Cited Medium: Internet ISSN: 1932-6203 (Electronic) Linking ISSN: 19326203 NLM ISO Abbreviation: PLoS One Subsets: MEDLINE

RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model.

Authors: Aimiuwu OV Fowler AM Sah M et al.

Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2020 Jul 08; Vol. 28 (7), pp. 1706-1716. Date of Electronic Publication: 2020 Apr 16.

Publication Type: Journal Article; Research Support, N.I.H., Extramural

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE

AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD.

Authors: Giesige CR Wallace LM Heller KN et al.

Source: JCI insight [JCI Insight] 2018 Nov 15; Vol. 3 (22). Date of Electronic Publication: 2018 Nov 15.

Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

Journal Info: Publisher: American Society for Clinical Investigation Country of Publication: United States NLM ID: 101676073 Publication Model: Electronic Cited Medium: Internet ISSN: 2379-3708 (Electronic) Linking ISSN: 23793708 NLM ISO Abbreviation: JCI Insight Subsets: MEDLINE

Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD.

Authors: Wallace LM Saad NY Pyne NK et al.

Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2017 Dec 24; Vol. 8, pp. 121-130. Date of Electronic Publication: 2017 Dec 24 (Print Publication: 2018).

Publication Type: Journal Article

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE