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المؤلفون: Long BR; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA. Electronic address: brian.long@bmrn.com., Robinson TM; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Day JRS; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Yu H; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Lau K; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Imtiaz U; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Patton KS; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., de Hart G; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Henshaw J; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Agarwal S; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Vettermann C; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Zoog SJ; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA., Gupta S; BioMarin Pharmaceutical Inc., Novato, CA 94949, USA.

المصدر: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Jul 03; Vol. 32 (7), pp. 2052-2063. Date of Electronic Publication: 2024 May 24.

نوع المنشور: Journal Article; Clinical Trial, Phase III

بيانات الدورية: 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

مواضيع طبية MeSH: Hemophilia A*/therapy , Hemophilia A*/immunology , Hemophilia A*/genetics , Dependovirus*/genetics , Dependovirus*/immunology , Genetic Therapy*/methods , Genetic Vectors*/genetics , Genetic Vectors*/administration & dosage , Factor VIII*/genetics , Factor VIII*/immunology, Humans ; Male ; Adult ; Treatment Outcome ; Transgenes ; Young Adult ; Antibodies, Viral/immunology ; Antibodies, Viral/blood ; Middle Aged

SCR Organism: Adeno-associated virus-5

المؤلفون: Chinn LK; Department of Psychology, Tulane University, 200 Broadway Street Suite 206, New Orleans, LA 70118, USA; Texas Institute for Measurement, Evaluation, and Statistics, University of Houston, Health 1, 4349 Martin Luther King Boulevard, Houston, TX 77204, USA. Electronic address: lisa.chinn@times.uh.edu., Noonan CF; Department of Psychology, Tulane University, 200 Broadway Street Suite 206, New Orleans, LA 70118, USA., Patton KS; Department of Psychology, Tulane University, 200 Broadway Street Suite 206, New Orleans, LA 70118, USA., Lockman JJ; Department of Psychology, Tulane University, 200 Broadway Street Suite 206, New Orleans, LA 70118, USA; Department of Human Development and Family Sciences, University of Texas at Austin, 108 E. Dean Keeton Street, Austin, TX 78712, USA.

المصدر: Current biology : CB [Curr Biol] 2024 Mar 25; Vol. 34 (6), pp. 1370-1375.e2. Date of Electronic Publication: 2024 Mar 04.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Cell Press Country of Publication: England NLM ID: 9107782 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1879-0445 (Electronic) Linking ISSN: 09609822 NLM ISO Abbreviation: Curr Biol Subsets: MEDLINE

مواضيع طبية MeSH: Touch* , Visual Perception*, Humans ; Infant ; Research Design

المؤلفون: Patton KS; Bioanalytical Sciences, BioMarin Pharmaceutical, 791 Lincoln Avenue, San Rafael, CA 94901, USA., Harrison MT; Immunology, Precision for Medicine, 2686 Middlefield Road, Redwood City, CA 94063, USA., Long BR; Bioanalytical Sciences, BioMarin Pharmaceutical, 791 Lincoln Avenue, San Rafael, CA 94901, USA., Lau K; Bioanalytical Sciences, BioMarin Pharmaceutical, 791 Lincoln Avenue, San Rafael, CA 94901, USA., Holcomb J; Bioanalytical Sciences, BioMarin Pharmaceutical, 791 Lincoln Avenue, San Rafael, CA 94901, USA., Owen R; Immunology, Precision for Medicine, 2686 Middlefield Road, Redwood City, CA 94063, USA., Kasprzyk T; Bioanalytical Sciences, BioMarin Pharmaceutical, 791 Lincoln Avenue, San Rafael, CA 94901, USA., Janetzki S; ZellNet Consulting, 555 North Avenue, Suite 25-S, Fort Lee, NJ 07024, USA., Zoog SJ; Bioanalytical Sciences, BioMarin Pharmaceutical, 791 Lincoln Avenue, San Rafael, CA 94901, USA., Vettermann C; Bioanalytical Sciences, BioMarin Pharmaceutical, 791 Lincoln Avenue, San Rafael, CA 94901, USA.

المصدر: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2021 May 29; Vol. 22, pp. 183-195. Date of Electronic Publication: 2021 May 29 (Print Publication: 2021).

نوع المنشور: Journal Article

بيانات الدورية: 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