Search Results - "Porteus MH"

Unveiling the cut-and-repair cycle of designer nucleases in human stem and T cells via CLEAR-time dPCR.

Authors: White N Chalk JA Hu YT et al.

Source: Nature communications [Nat Commun] 2025 Nov 03; Vol. 16 (1), pp. 9571. Date of Electronic Publication: 2025 Nov 03.

Publication Type: Journal Article

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

Multilayered HIV-1 resistance in HSPCs through CCR5 Knockout and B cell secretion of HIV-inhibiting antibodies.

Authors: Feist WN Luna SE Ben-Efraim K et al.

Source: Nature communications [Nat Commun] 2025 Apr 01; Vol. 16 (1), pp. 3103. Date of Electronic Publication: 2025 Apr 01.

Publication Type: Journal Article

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

Novel humanized loss-of-function NF1 mouse model of juvenile myelomonocytic leukemia.

Authors: Sinha R Patil RV Romano R et al.

Source: Blood advances [Blood Adv] 2026 Feb 10; Vol. 10 (3), pp. 837-849.

Publication Type: Journal Article

Journal Info: Publisher: American Society of Hematology Country of Publication: United States NLM ID: 101698425 Publication Model: Print Cited Medium: Internet ISSN: 2473-9537 (Electronic) Linking ISSN: 24739529 NLM ISO Abbreviation: Blood Adv Subsets: MEDLINE

Engineering synthetic signaling receptors to enable erythropoietin-free erythropoiesis.

Authors: Shah AP Majeti KR Ekman FK et al.

Source: Nature communications [Nat Commun] 2025 Jan 29; Vol. 16 (1), pp. 1140. Date of Electronic Publication: 2025 Jan 29.

Publication Type: Journal Article

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

Discovery of a pre-vein progenitor that requires VEGF/ERK inhibition to complete vein differentiation.

Authors: Ang LT Zheng SL Liu KJ et al.

Source: BioRxiv : the preprint server for biology [bioRxiv] 2025 Dec 04. Date of Electronic Publication: 2025 Dec 04.

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

Cell and gene therapy accessibility.

Authors: Rouce RH Porteus MH

Source: Science (New York, N.Y.) [Science] 2024 Aug 02; Vol. 385 (6708), pp. 475. Date of Electronic Publication: 2024 Aug 01.

Publication Type: Editorial

Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 0404511 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1095-9203 (Electronic) Linking ISSN: 00368075 NLM ISO Abbreviation: Science Subsets: MEDLINE

Rewiring endogenous genes in CAR T cells for tumour-restricted payload delivery.

Authors: Chen AXY Yap KM Kim JS et al.

Source: Nature [Nature] 2025 Aug; Vol. 644 (8075), pp. 241-251. Date of Electronic Publication: 2025 Jul 02.

Publication Type: Journal Article

Journal Info: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 0410462 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-4687 (Electronic) Linking ISSN: 00280836 NLM ISO Abbreviation: Nature Subsets: MEDLINE

A Reset for Bioethics: A Statement from the Global Observatory for Genome Editing.

Authors: Jasanoff S Hurlbut JB Saha K et al.

Source: The CRISPR journal [CRISPR J] 2025 Aug; Vol. 8 (4), pp. 232-238. Date of Electronic Publication: 2025 May 22.

Publication Type: Journal Article

Journal Info: Publisher: Mary Ann Liebert, Inc Country of Publication: United States NLM ID: 101738191 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2573-1602 (Electronic) Linking ISSN: 25731599 NLM ISO Abbreviation: CRISPR J Subsets: MEDLINE

Gene regulation in inborn errors of immunity: Implications for gene therapy design and efficacy.

Authors: Ghanim HY Porteus MH

Source: Immunological reviews [Immunol Rev] 2024 Mar; Vol. 322 (1), pp. 157-177. Date of Electronic Publication: 2024 Jan 17.

Publication Type: Journal Article; Review

Journal Info: Publisher: Blackwell Country of Publication: England NLM ID: 7702118 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1600-065X (Electronic) Linking ISSN: 01052896 NLM ISO Abbreviation: Immunol Rev Subsets: MEDLINE

Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cells.

Authors: Baik R Cromer MK Glenn SE et al.

Source: Nature communications [Nat Commun] 2024 Jan 02; Vol. 15 (1), pp. 111. Date of Electronic Publication: 2024 Jan 02.

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

A differentiated β-globin gene replacement strategy uses heterologous introns to restore physiological expression.

Authors: Wallace KA Gerstenberg TL Ennis CL et al.

Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Apr 02; Vol. 33 (4), pp. 1407-1419. Date of Electronic Publication: 2025 Feb 28.

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

Combined lineage tracing and scRNA-seq reveals unexpected first heart field predominance of human iPSC differentiation.

Authors: Galdos FX Lee C Lee S et al.

Source: ELife [Elife] 2023 Jun 07; Vol. 12. Date of Electronic Publication: 2023 Jun 07.

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

Journal Info: Publisher: eLife Sciences Publications, Ltd Country of Publication: England NLM ID: 101579614 Publication Model: Electronic Cited Medium: Internet ISSN: 2050-084X (Electronic) Linking ISSN: 2050084X NLM ISO Abbreviation: Elife Subsets: MEDLINE

Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells.

Authors: Chu SN Soupene E Sharma D et al.

Source: Cell reports [Cell Rep] 2025 Jan 28; Vol. 44 (1), pp. 115141. Date of Electronic Publication: 2025 Jan 03.

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

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101573691 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2211-1247 (Electronic) NLM ISO Abbreviation: Cell Rep Subsets: MEDLINE

Durable reconstitution of sinonasal epithelium by transplant of CFTR gene corrected airway stem cells.

Authors: Bravo DT Vaidyanathan S Baker J et al.

Source: BioRxiv : the preprint server for biology [bioRxiv] 2025 Jan 26. Date of Electronic Publication: 2025 Jan 26.

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

Human striatal progenitor cells that contain inducible safeguards and overexpress BDNF rescue Huntington's disease phenotypes.

Authors: Simmons DA Selvaraj S Chen T et al.

Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2025 Jan 22; Vol. 33 (1), pp. 101415. Date of Electronic Publication: 2025 Jan 22 (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

CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells.

Authors: Brault J Liu T Liu S et al.

Source: Frontiers in immunology [Front Immunol] 2023 Jan 04; Vol. 13, pp. 1067417. Date of Electronic Publication: 2023 Jan 04 (Print Publication: 2022).

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

Journal Info: Publisher: Frontiers Research Foundation] Country of Publication: Switzerland NLM ID: 101560960 Publication Model: eCollection Cited Medium: Internet ISSN: 1664-3224 (Electronic) Linking ISSN: 16643224 NLM ISO Abbreviation: Front Immunol Subsets: MEDLINE

Failure of metabolic checkpoint control during late-stage granulopoiesis drives neutropenia in reticular dysgenesis.

Authors: Wang W Arreola M Mathews T et al.

Source: Blood [Blood] 2024 Dec 26; Vol. 144 (26), pp. 2718-2734.

Publication Type: Journal Article

Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 7603509 Publication Model: Print Cited Medium: Internet ISSN: 1528-0020 (Electronic) Linking ISSN: 00064971 NLM ISO Abbreviation: Blood Subsets: MEDLINE

Human assembloids reveal the consequences of CACNA1G gene variants in the thalamocortical pathway.

Authors: Kim JI Miura Y Li MY et al.

Source: Neuron [Neuron] 2024 Dec 18; Vol. 112 (24), pp. 4048-4059.e7. Date of Electronic Publication: 2024 Oct 16.

Publication Type: Journal Article

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 8809320 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-4199 (Electronic) Linking ISSN: 08966273 NLM ISO Abbreviation: Neuron Subsets: MEDLINE

Enhancement of erythropoietic output by Cas9-mediated insertion of a natural variant in haematopoietic stem and progenitor cells.

Authors: Luna SE Camarena J Hampton JP et al.

Source: Nature biomedical engineering [Nat Biomed Eng] 2024 Dec; Vol. 8 (12), pp. 1540-1552. Date of Electronic Publication: 2024 Jun 17.

Publication Type: Journal Article

Journal Info: Publisher: Springer Nature Country of Publication: England NLM ID: 101696896 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2157-846X (Electronic) Linking ISSN: 2157846X NLM ISO Abbreviation: Nat Biomed Eng Subsets: MEDLINE

TET2 regulates early and late transitions in exhausted CD8⁺ T cell differentiation and limits CAR T cell function.

Authors: Dimitri AJ Baxter AE Chen GM et al.

Source: Science advances [Sci Adv] 2024 Nov 15; Vol. 10 (46), pp. eadp9371. Date of Electronic Publication: 2024 Nov 13.

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

Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101653440 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2375-2548 (Electronic) Linking ISSN: 23752548 NLM ISO Abbreviation: Sci Adv Subsets: MEDLINE