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Academic Journal

Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency.

  • Authors : Pham V; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Human Genetics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.

Subjects: Genetic Therapy*/Genetic Therapy*/Genetic Therapy*/methods ; Disease Models, Animal* ; Multiple Sulfatase Deficiency Disease*/Multiple Sulfatase Deficiency Disease*/Multiple Sulfatase Deficiency Disease*/therapy

  • Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Nov 06; Vol. 32 (11), pp. 3829-3846. Date of Electronic Publication: 2024 Aug 22.Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited

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Academic Journal

Secondary failure of lentiviral vector gene therapy in a cerebral adrenoleukodystrophy patient with an ABCD1 whole-gene deletion.

  • Authors : Lund TC; Department of Pediatrics, Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA. Electronic address: .; Orchard PJ

Subjects: Adrenoleukodystrophy*/Adrenoleukodystrophy*/Adrenoleukodystrophy*/therapy ; Adrenoleukodystrophy*/Adrenoleukodystrophy*/Adrenoleukodystrophy*/genetics ; ATP Binding Cassette Transporter, Subfamily D, Member 1*/ATP Binding Cassette Transporter, Subfamily D, Member 1*/ATP Binding Cassette Transporter, Subfamily D, Member 1*/genetics

  • Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Oct 02; Vol. 32 (10), pp. 3313-3317. Date of Electronic Publication: 2024 Aug 05.Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited

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Academic Journal

Protocol for transducing human primary epithelial prostate cells and patient-derived organoids with high efficiency.

  • Authors : Jobin C; Department of Molecular Medicine, Université Laval, Quebec City, QC, Canada; Endocrinology and Nephrology Division, CHU de Québec-Université Laval Research Center (CRCHUQ-UL), Quebec City, QC, Canada

Subjects: Organoids*/Organoids*/Organoids*/cytology ; Organoids*/Organoids*/Organoids*/metabolism ; Prostate*/Prostate*/Prostate*/cytology

  • Source: STAR protocols [STAR Protoc] 2024 Sep 20; Vol. 5 (3), pp. 103200. Date of Electronic Publication: 2024 Jul 18.Publisher: Cell Press Country of Publication: United States NLM ID: 101769501 Publication Model: Print-Electronic Cited

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Academic Journal

Modified lentiviral globin gene therapy for pediatric β 0 /β 0 transfusion-dependent β-thalassemia: A single-center, single-arm pilot trial.

  • Authors : Li S; 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China, Kunming, Yunnan 650100, China.; Ling S

Subjects: beta-Thalassemia*/beta-Thalassemia*/beta-Thalassemia*/therapy ; beta-Thalassemia*/beta-Thalassemia*/beta-Thalassemia*/genetics ; Genetic Therapy*

  • Source: Cell stem cell [Cell Stem Cell] 2024 Jul 05; Vol. 31 (7), pp. 961-973.e8. Date of Electronic Publication: 2024 May 16.Publisher: Cell Press Country of Publication: United States NLM ID: 101311472 Publication Model: Print-Electronic Cited

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Academic Journal

Analyzing efficiency of a lentiviral shRNA knockdown system in human enteroids using western blot and flow cytometry.

  • Authors : Wilson AP; Pediatrics, University of Oklahoma Health Sciences Center, Oklahoma City, OK 73104, USA. Electronic address: .; Moshal KS

Subjects: Lentivirus*/Lentivirus*/Lentivirus*/genetics ; Flow Cytometry*/Flow Cytometry*/Flow Cytometry*/methods ; Gene Knockdown Techniques*/Gene Knockdown Techniques*/Gene Knockdown Techniques*/methods

  • Source: STAR protocols [STAR Protoc] 2024 Jun 21; Vol. 5 (2), pp. 103082. Date of Electronic Publication: 2024 May 22.Publisher: Cell Press Country of Publication: United States NLM ID: 101769501 Publication Model: Print-Electronic Cited

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Academic Journal

Lentiviral expression of wild-type LAMA3A restores cell adhesion in airway basal cells from children with epidermolysis bullosa.

  • Authors : Lau CH; Epithelial Cell Biology in ENT Research (EpiCENTR) Group, UCL Great Ormond Street Institute of Child Health, University College London, 20c Guilford Street, London WC1N 1DZ, UK.; Rouhani MJ

Subjects: Laminin*/Laminin*/Laminin*/metabolism ; Laminin*/Laminin*/Laminin*/genetics ; Cell Adhesion*

  • Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 May 01; Vol. 32 (5), pp. 1497-1509. Date of Electronic Publication: 2024 Feb 29.Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited

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Academic Journal

Envelope protein-specific B cell receptors direct lentiviral vector tropism in vivo.

  • Authors : Takano KA; Division of Hematology and Oncology, Department of Medicine, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA; UCLA AIDS Institute, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.

Subjects: Lentivirus*/Lentivirus*/Lentivirus*/genetics ; Receptors, Antigen, B-Cell*/Receptors, Antigen, B-Cell*/Receptors, Antigen, B-Cell*/metabolism ; Receptors, Antigen, B-Cell*/Receptors, Antigen, B-Cell*/Receptors, Antigen, B-Cell*/genetics

  • Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 May 01; Vol. 32 (5), pp. 1311-1327. Date of Electronic Publication: 2024 Mar 06.Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited

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