Search Results - "Kohn DB"

The enhanced green fluorescent protein (eGFP) is minimally immunogenic in C57BL/6 mice.

Authors: Skelton, D Satake, N Kohn, DB

Source: Gene Therapy. Dec2001, Vol. 8 Issue 23, p1813. 2p.

Clinical outcomes of lentiviral vector gene therapy for sickle cell disease.

Authors: Prueksapraopong C Fernandes A Campo Fernandez B et al.

Source: Blood advances [Blood Adv] 2026 Jul 28; Vol. 10 (14), pp. 4991-5002.

Publication Type: Journal Article; Clinical Trial, Phase I; Clinical Trial, Phase II

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

Newborn screening reduces survival disparities in SCID after stem cell transplant: A PIDTC report.

Authors: Winestone LE Logan BR Liu X et al.

Source: Journal of human immunity [J Hum Immun] 2026 Jul 07; Vol. 2 (5), pp. e20250231. Date of Electronic Publication: 2026 Jul 07 (Print Publication: 2026).

Publication Type: Journal Article

Journal Info: Publisher: Rockefeller University Press Country of Publication: United States NLM ID: 9919054337406676 Publication Model: eCollection Cited Medium: Internet ISSN: 3065-8993 (Electronic) Linking ISSN: 30658993 NLM ISO Abbreviation: J Hum Immun Subsets: PubMed not MEDLINE

Response to "Defining durability in SCD gene therapy".

Authors: Kohn DB Prueksapraopong C

Source: Blood advances [Blood Adv] 2026 Jun 23. Date of Electronic Publication: 2026 Jun 23.

Publication Type: Journal Article

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

Long-term stability of posttranscriptional genetic silencing of BCL11A using a shmiR vector in Sickle Cell Disease.

Authors: Esrick EB Lehmann L Federico A et al.

Source: Blood [Blood] 2026 Jun 02. Date of Electronic Publication: 2026 Jun 02.

Publication Type: Journal Article

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

Human cancer-targeted immunity via transgenic hematopoietic stem cell progeny.

Authors: Nowicki TS Deen NNA Peters CW et al.

Source: Nature communications [Nat Commun] 2025 Jul 01; Vol. 16 (1), pp. 5599. Date of Electronic Publication: 2025 Jul 01.

Publication Type: Journal Article; Clinical Trial, Phase I

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 blueprint to accelerate rare pediatric gene therapy approvals.

Authors: Abou-El-Enein M Walters MC Sanghavi D et al.

Source: Nature medicine [Nat Med] 2026 Apr; Vol. 32 (4), pp. 1170-1171.

Publication Type: Letter

Journal Info: Publisher: Nature Publishing Company Country of Publication: United States NLM ID: 9502015 Publication Model: Print Cited Medium: Internet ISSN: 1546-170X (Electronic) Linking ISSN: 10788956 NLM ISO Abbreviation: Nat Med Subsets: MEDLINE; In Process

Prime Editing for p47^phox-Deficient Chronic Granulomatous Disease.

Authors: Gori JL Haddad E Frangoul H et al.

Source: The New England journal of medicine [N Engl J Med] 2026 Mar 26; Vol. 394 (12), pp. 1195-1203. Date of Electronic Publication: 2025 Dec 07.

Publication Type: Clinical Trial, Phase I; Clinical Trial, Phase II; Journal Article; Multicenter Study

Journal Info: Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE

Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report.

Authors: Alexander JL Dávila Saldaña BJ Brazauskas R et al.

Source: Blood advances [Blood Adv] 2026 Mar 10; Vol. 10 (5), pp. 1783-1798.

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

Outcomes following matched sibling donor transplantation for severe combined immunodeficiency: a report from the PIDTC.

Authors: Rayes A Logan BR Liu X et al.

Source: Blood advances [Blood Adv] 2026 Feb 10; Vol. 10 (3), pp. 887-900.

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

Clinical spectrum of Wiskott-Aldrich syndrome carriers: Self-reported survey of 193 carriers.

Authors: Chandrakasan S Westbrook A Griffith LM et al.

Source: Clinical immunology (Orlando, Fla.) [Clin Immunol] 2026 Feb; Vol. 283, pp. 110658. Date of Electronic Publication: 2025 Dec 17.

Publication Type: Journal Article

Journal Info: Publisher: Academic Press Country of Publication: United States NLM ID: 100883537 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1521-7035 (Electronic) Linking ISSN: 15216616 NLM ISO Abbreviation: Clin Immunol Subsets: MEDLINE

Umbilical Cord Blood Transplantation Provides an Alternative for Patients With Chronic Granulomatous Disease Lacking HLA-Matched Donors: A PIDTC Report.

Authors: Arnold DE Leiding JW Logan B et al.

Source: Transplantation and cellular therapy [Transplant Cell Ther] 2026 Jan; Vol. 32 (1), pp. 89.e1-89.e10. Date of Electronic Publication: 2025 Oct 02.

Publication Type: Journal Article

Journal Info: Publisher: Elsevier Inc Country of Publication: United States NLM ID: 101774629 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2666-6367 (Electronic) Linking ISSN: 26666367 NLM ISO Abbreviation: Transplant Cell Ther Subsets: MEDLINE

Neurodevelopmental outcomes following hematopoietic cell transplantation for patients with severe combined immunodeficiency (SCID): A PIDTC study.

Authors: Brumm VL Kidd SA Logan BR et al.

Source: Journal of human immunity [J Hum Immun] 2025 Dec 17; Vol. 2 (1), pp. e20250163. Date of Electronic Publication: 2025 Dec 17 (Print Publication: 2026).

Publication Type: Journal Article

Journal Info: Publisher: Rockefeller University Press Country of Publication: United States NLM ID: 9919054337406676 Publication Model: eCollection Cited Medium: Internet ISSN: 3065-8993 (Electronic) Linking ISSN: 30658993 NLM ISO Abbreviation: J Hum Immun Subsets: PubMed not MEDLINE

TriLeukeVax: A CD80/IL-15/IL-15Rα Expressing Autologous AML Cell Vaccine Elicits Robust Anti-Leukemic Cytolytic Activity.

Authors: Du J Wijayaratna UN Wang X et al.

Source: BioRxiv : the preprint server for biology [bioRxiv] 2025 Nov 13. Date of Electronic Publication: 2025 Nov 13.

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

Lentiviral vectors for hematopoietic stem cell gene therapy restore α-globin expression in α-thalassemia red blood cells.

Authors: Segura EER Hart K Campo Fernandez B et al.

Source: Cell reports. Medicine [Cell Rep Med] 2025 Oct 21; Vol. 6 (10), pp. 102362. Date of Electronic Publication: 2025 Sep 17.

Publication Type: Journal Article

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101766894 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2666-3791 (Electronic) Linking ISSN: 26663791 NLM ISO Abbreviation: Cell Rep Med Subsets: MEDLINE

Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency.

Authors: Booth C Masiuk K Vazouras K et al.

Source: The New England journal of medicine [N Engl J Med] 2025 Oct 16; Vol. 393 (15), pp. 1486-1497.

Publication Type: Journal Article; Multicenter Study; Observational Study

Journal Info: Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE

Quantifying the mutational landscape of retroviral and lentiviral vectors in gene therapy patients.

Authors: Hart KL Crisostomo RV Mittelhauser A et al.

Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Sep 03; Vol. 33 (9), pp. 4459-4470. Date of Electronic Publication: 2025 May 30.

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

Hematopoietic stem cell gene therapy for the treatment of X-linked agammaglobulinemia.

Authors: Luthers CR Mittelhauser A Colamartino A et al.

Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2025 Aug 12; Vol. 33 (3), pp. 101555. Date of Electronic Publication: 2025 Aug 12 (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: In Process

Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment.

Authors: Keller MD Hanley PJ Chi YY et al.

Source: Nature communications [Nat Commun] 2024 Apr 18; Vol. 15 (1), pp. 3258. Date of Electronic Publication: 2024 Apr 18.

Publication Type: Multicenter Study; Clinical Trial, Phase II; 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

Gene therapy for adenosine deaminase severe combined immune deficiency-An unexpected journey of four decades.

Authors: Kohn DB; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, California, USA.; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, California, USA.; Department of Molecular & Medical Pharmacology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, California, USA.

Source: Immunological reviews [Immunol Rev] 2024 Mar; Vol. 322 (1), pp. 148-156. Date of Electronic Publication: 2023 Nov 30.

Publication Type: Journal Article; Review; Research Support, Non-U.S. Gov't

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