Search Results - "Lagor WR"

Cytosine base editing of LPA in transgenic mice averts large deletions.

Authors: Chuecos MA Park SH Bhakta MM et al.

Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2026 Jun 03; Vol. 34 (6), pp. 3334-3352. Date of Electronic Publication: 2026 Mar 03.

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

Transient YAP activation uncovers the neurogenic potential of proliferative mammalian Müller glia.

Authors: Laserna EJ Saltykova IV Hall BM et al.

Source: PNAS nexus [PNAS Nexus] 2026 May 28; Vol. 5 (6), pp. pgag188. Date of Electronic Publication: 2026 May 28 (Print Publication: 2026).

Publication Type: Journal Article

Journal Info: Publisher: Oxford University Press on behalf of the National Academy of Sciences Country of Publication: England NLM ID: 9918367777906676 Publication Model: eCollection Cited Medium: Internet ISSN: 2752-6542 (Electronic) Linking ISSN: 27526542 NLM ISO Abbreviation: PNAS Nexus Subsets: PubMed not MEDLINE

In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene.

Authors: De Giorgi M Park SH Castoreno A et al.

Source: Science translational medicine [Sci Transl Med] 2025 Feb 12; Vol. 17 (785), pp. eadk3920. Date of Electronic Publication: 2025 Feb 12.

Publication Type: Journal Article

Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE

Liver directed adeno-associated viral vectors to treat metabolic disease.

Authors: Chuecos MA Lagor WR

Source: Journal of inherited metabolic disease [J Inherit Metab Dis] 2024 Jan; Vol. 47 (1), pp. 22-40. Date of Electronic Publication: 2023 Jun 05.

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

Journal Info: Publisher: Wiley Country of Publication: United States NLM ID: 7910918 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1573-2665 (Electronic) Linking ISSN: 01418955 NLM ISO Abbreviation: J Inherit Metab Dis Subsets: MEDLINE

Targeting AAV vectors to the central nervous system by engineering capsid-receptor interactions that enable crossing of the blood-brain barrier.

Authors: Huang Q Chen AT Chan KY et al.

Source: PLoS biology [PLoS Biol] 2023 Jul 19; Vol. 21 (7), pp. e3002112. Date of Electronic Publication: 2023 Jul 19 (Print Publication: 2023).

Publication Type: Journal Article

Journal Info: Publisher: Public Library of Science Country of Publication: United States NLM ID: 101183755 Publication Model: eCollection Cited Medium: Internet ISSN: 1545-7885 (Electronic) Linking ISSN: 15449173 NLM ISO Abbreviation: PLoS Biol Subsets: MEDLINE

Repair Drive improves gene editing in the liver.

Authors: De Giorgi M Park SH Bao G et al.

Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Apr 02; Vol. 33 (4), pp. 1324-1326. Date of Electronic Publication: 2025 Mar 19.

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; In Process

A comprehensive atlas of AAV tropism in the mouse.

Authors: Walkey CJ Snow KJ Bulcha J et al.

Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Mar 05; Vol. 33 (3), pp. 1282-1299. Date of Electronic Publication: 2025 Jan 25.

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

MBNL overexpression rescues cardiac phenotypes in a myotonic dystrophy type 1 heart mouse model.

Authors: Hu RC Zhang Y Nitschke L et al.

Source: The Journal of clinical investigation [J Clin Invest] 2025 Feb 11; Vol. 135 (7). Date of Electronic Publication: 2025 Feb 11.

Publication Type: Journal Article

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

Rescue of glutaric aciduria type I in mice by liver-directed therapies.

Authors: Barzi M Johnson CG Chen T et al.

Source: Science translational medicine [Sci Transl Med] 2023 Apr 19; Vol. 15 (692), pp. eadf4086. Date of Electronic Publication: 2023 Apr 19.

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

Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE

Adeno-Associated virus 8 delivers an immunomodulatory peptide to mouse liver more efficiently than to rat liver.

Authors: Wang Y Hurley A De Giorgi M et al.

Source: PloS one [PLoS One] 2023 Apr 11; Vol. 18 (4), pp. e0283996. Date of Electronic Publication: 2023 Apr 11 (Print Publication: 2023).

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

Treating Cardiovascular Disease with Liver Genome Engineering.

Authors: Hurley A Lagor WR

Source: Current atherosclerosis reports [Curr Atheroscler Rep] 2022 Feb; Vol. 24 (2), pp. 75-84. Date of Electronic Publication: 2022 Mar 01.

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

Journal Info: Publisher: Springer Country of Publication: United States NLM ID: 100897685 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1534-6242 (Electronic) Linking ISSN: 15233804 NLM ISO Abbreviation: Curr Atheroscler Rep Subsets: MEDLINE

A Comprehensive Atlas of AAV Tropism in the Mouse.

Authors: Walkey CJ Snow KJ Bulcha J et al.

Source: BioRxiv : the preprint server for biology [bioRxiv] 2024 Sep 10. Date of Electronic Publication: 2024 Sep 10.

Publication Type: Journal Article; Preprint

Journal Info: Country of Publication: United States NLM ID: 101680187 Publication Model: Electronic Cited Medium: Internet ISSN: 2692-8205 (Electronic) Linking ISSN: 26928205 NLM ISO Abbreviation: bioRxiv Subsets: PubMed not MEDLINE

Less is more: Allele-specific removal of dysfunctional RYR1 channel subunits.

Authors: Sun D Lagor WR

Source: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2024 Aug 30; Vol. 35 (3), pp. 102301. Date of Electronic Publication: 2024 Aug 30 (Print Publication: 2024).

Publication Type: News

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101581621 Publication Model: eCollection Cited Medium: Print ISSN: 2162-2531 (Print) Linking ISSN: 21622531 NLM ISO Abbreviation: Mol Ther Nucleic Acids Subsets: PubMed not MEDLINE

Simple improvements in vector design afford substantial gains in AAV delivery of aggregation-slowing Aβ variants.

Authors: Borgenheimer E Trueblood C Nguyen BL et al.

Source: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2024 Aug 26; Vol. 35 (4), pp. 102314. Date of Electronic Publication: 2024 Aug 26 (Print Publication: 2024).

Publication Type: Journal Article

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101581621 Publication Model: eCollection Cited Medium: Print ISSN: 2162-2531 (Print) Linking ISSN: 21622531 NLM ISO Abbreviation: Mol Ther Nucleic Acids Subsets: PubMed not MEDLINE

Long-term efficacy and safety of cardiac genome editing for catecholaminergic polymorphic ventricular tachycardia.

Authors: Moore OM Aguilar-Sanchez Y Lahiri SK et al.

Source: The journal of cardiovascular aging [J Cardiovasc Aging] 2024 Jan; Vol. 4 (1). Date of Electronic Publication: 2024 Jan 04.

Publication Type: Journal Article

Journal Info: Publisher: OAE Publishing Country of Publication: United States NLM ID: 101778645 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2768-5993 (Electronic) Linking ISSN: 27685993 NLM ISO Abbreviation: J Cardiovasc Aging Subsets: PubMed not MEDLINE

High levels of lipoprotein(a) in transgenic mice exacerbate atherosclerosis and promote vulnerable plaque features in a sex-specific manner.

Authors: Assini JM Clark JR Youssef A et al.

Source: Atherosclerosis [Atherosclerosis] 2023 Nov; Vol. 384, pp. 117150. Date of Electronic Publication: 2023 May 30.

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

Journal Info: Publisher: Elsevier Country of Publication: Ireland NLM ID: 0242543 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1879-1484 (Electronic) Linking ISSN: 00219150 NLM ISO Abbreviation: Atherosclerosis Subsets: MEDLINE

Speg interactions that regulate the stability of excitation-contraction coupling protein complexes in triads and dyads.

Authors: Lee CS Jung SY Yee RSZ et al.

Source: Communications biology [Commun Biol] 2023 Sep 14; Vol. 6 (1), pp. 942. Date of Electronic Publication: 2023 Sep 14.

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

Journal Info: Publisher: Nature Publishing Group UK Country of Publication: England NLM ID: 101719179 Publication Model: Electronic Cited Medium: Internet ISSN: 2399-3642 (Electronic) Linking ISSN: 23993642 NLM ISO Abbreviation: Commun Biol Subsets: MEDLINE

TFEB regulates murine liver cell fate during development and regeneration.

Authors: Pastore N Huynh T Herz NJ et al.

Source: Nature communications [Nat Commun] 2020 May 18; Vol. 11 (1), pp. 2461. Date of Electronic Publication: 2020 May 18.

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

Journal Info: Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE

In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene.

Authors: De Giorgi M Park SH Castoreno A et al.

Source: BioRxiv : the preprint server for biology [bioRxiv] 2023 Jul 29. Date of Electronic Publication: 2023 Jul 29.

Publication Type: Preprint; Journal Article

Journal Info: Country of Publication: United States NLM ID: 101680187 Publication Model: Electronic Cited Medium: Internet ISSN: 2692-8205 (Electronic) Linking ISSN: 26928205 NLM ISO Abbreviation: bioRxiv Subsets: PubMed not MEDLINE

In vivo editing of the pan-endothelium by immunity evading simian adenoviral vector.

Authors: Lorincz R Alvarez AB Walkey CJ et al.

Source: Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie [Biomed Pharmacother] 2023 Feb; Vol. 158, pp. 114189. Date of Electronic Publication: 2022 Dec 30.

Publication Type: Journal Article

Journal Info: Publisher: Editions Scientifiques Elsevier Country of Publication: France NLM ID: 8213295 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1950-6007 (Electronic) Linking ISSN: 07533322 NLM ISO Abbreviation: Biomed Pharmacother Subsets: MEDLINE