Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.

Autor: Shi X; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AG&M, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands.; School of Life Sciences, Liaoning Provincial Key Laboratory of Biotechnology and Drug Discovery, Liaoning Normal University, Dalian 116082, P.R. China., Bortolussi G; International Centre for Genetic Engineering and Biotechnology (ICGEB), Padriciano 99, 34149 Trieste, Italy., Collaud F; Genethon, 91000 Evry, France.; Université Paris-Saclay, Université d'Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France., Lebrun PR; Genethon, 91000 Evry, France., Bloemendaal LT; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AG&M, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., Guerchet N; Genethon, 91000 Evry, France., Rudi de Waart D; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AG&M, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., Sellier P; Genethon, 91000 Evry, France.; Université Paris-Saclay, Université d'Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France., Duijst S; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AG&M, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., Veron P; Genethon, 91000 Evry, France., Mingozzi F; Genethon, 91000 Evry, France., Kishimoto TK; Selecta Biosciences, Watertown, MA 02472, USA., Ronzitti G; Genethon, 91000 Evry, France.; Université Paris-Saclay, Université d'Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France., Bosma P; Amsterdam UMC, University of Amsterdam, Tytgat Institute for Liver and Intestinal Research, AG&M, Meibergdreef 69-71, 1105 BK Amsterdam, the Netherlands., Muro AF; International Centre for Genetic Engineering and Biotechnology (ICGEB), Padriciano 99, 34149 Trieste, Italy.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2024 Oct 28; Vol. 32 (4), pp. 101363. Date of Electronic Publication: 2024 Oct 28 (Print Publication: 2024).

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders.

Autor: El Andari J; Medical Faculty, Department of Infectious Diseases/Virology, Section Viral Vector Technologies, Cluster of Excellence CellNetworks, University of Heidelberg, 69120 Heidelberg, Germany.; BioQuant, University of Heidelberg, 69120 Heidelberg, Germany., Renaud-Gabardos E; Genethon, 91000 Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France., Tulalamba W; Department of Gene Therapy and Regenerative Medicine, Vrije Universiteit Brussel (VUB), Brussels 1090, Belgium., Weinmann J; Medical Faculty, Department of Infectious Diseases/Virology, Section Viral Vector Technologies, Cluster of Excellence CellNetworks, University of Heidelberg, 69120 Heidelberg, Germany.; BioQuant, University of Heidelberg, 69120 Heidelberg, Germany., Mangin L; Genethon, 91000 Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France., Pham QH; Department of Gene Therapy and Regenerative Medicine, Vrije Universiteit Brussel (VUB), Brussels 1090, Belgium., Hille S; University Hospital Schleswig-Holstein, Campus Kiel, Innere Medizin III, 24105 Kiel, Germany.; German Center for Cardiovascular Research (DZHK), partner site Hamburg/Kiel/Lübeck, Kiel, Germany., Bennett A; Department of Biochemistry and Molecular Biology, Center for Structural Biology, McKnight Brain Institute, University of Florida, Gainesville, FL 32610, USA., Attebi E; Genethon, 91000 Evry, France., Bourges E; Genethon, 91000 Evry, France., Leborgne C; Genethon, 91000 Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France., Guerchet N; Genethon, 91000 Evry, France., Fakhiri J; Medical Faculty, Department of Infectious Diseases/Virology, Section Viral Vector Technologies, Cluster of Excellence CellNetworks, University of Heidelberg, 69120 Heidelberg, Germany.; BioQuant, University of Heidelberg, 69120 Heidelberg, Germany., Krämer C; Medical Faculty, Department of Infectious Diseases/Virology, Section Viral Vector Technologies, Cluster of Excellence CellNetworks, University of Heidelberg, 69120 Heidelberg, Germany.; BioQuant, University of Heidelberg, 69120 Heidelberg, Germany., Wiedtke E; Medical Faculty, Department of Infectious Diseases/Virology, Section Viral Vector Technologies, Cluster of Excellence CellNetworks, University of Heidelberg, 69120 Heidelberg, Germany.; BioQuant, University of Heidelberg, 69120 Heidelberg, Germany., McKenna R; Department of Biochemistry and Molecular Biology, Center for Structural Biology, McKnight Brain Institute, University of Florida, Gainesville, FL 32610, USA., Guianvarc'h L; Genethon, 91000 Evry, France., Toueille M; Genethon, 91000 Evry, France., Ronzitti G; Genethon, 91000 Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France., Hebben M; Genethon, 91000 Evry, France., Mingozzi F; Genethon, 91000 Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France., VandenDriessche T; Department of Gene Therapy and Regenerative Medicine, Vrije Universiteit Brussel (VUB), Brussels 1090, Belgium.; Center for Molecular and Vascular Biology, Department of Cardiovascular Sciences, University of Leuven, Leuven 3000, Belgium., Agbandje-McKenna M; Department of Biochemistry and Molecular Biology, Center for Structural Biology, McKnight Brain Institute, University of Florida, Gainesville, FL 32610, USA., Müller OJ; University Hospital Schleswig-Holstein, Campus Kiel, Innere Medizin III, 24105 Kiel, Germany.; German Center for Cardiovascular Research (DZHK), partner site Hamburg/Kiel/Lübeck, Kiel, Germany., Chuah MK; Department of Gene Therapy and Regenerative Medicine, Vrije Universiteit Brussel (VUB), Brussels 1090, Belgium.; Center for Molecular and Vascular Biology, Department of Cardiovascular Sciences, University of Leuven, Leuven 3000, Belgium., Buj-Bello A; Genethon, 91000 Evry, France.; Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, 91000 Evry, France., Grimm D; Medical Faculty, Department of Infectious Diseases/Virology, Section Viral Vector Technologies, Cluster of Excellence CellNetworks, University of Heidelberg, 69120 Heidelberg, Germany.; BioQuant, University of Heidelberg, 69120 Heidelberg, Germany.; German Center for Infection Research (DZIF) and German Center for Cardiovascular Research (DZHK), partner site Heidelberg, Heidelberg, Germany.

Publikováno v: Science advances [Sci Adv] 2022 Sep 23; Vol. 8 (38), pp. eabn4704. Date of Electronic Publication: 2022 Sep 21.

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.

Autor: Colella P; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France. Electronic address: pcolella@stanford.edu., Sellier P; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Gomez MJ; Bioinformatics Unit, CNIC, Madrid, Spain., Biferi MG; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France., Tanniou G; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Guerchet N; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Cohen-Tannoudji M; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France., Moya-Nilges M; Pasteur Institute, Paris, France., van Wittenberghe L; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Daniele N; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Gjata B; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Krijnse-Locker J; Pasteur Institute, Paris, France., Collaud F; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Simon-Sola M; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Charles S; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Cagin U; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France., Mingozzi F; INTEGRARE, Genethon, Inserm, Univ Evry, Université Paris Saclay, Evry, France; University Pierre and Marie Curie Paris 6 and INSERM U974, Paris, France; Spark Therapeutics, Philadelphia, PA, USA. Electronic address: federico.mingozzi@sparktx.com.

Publikováno v: EBioMedicine [EBioMedicine] 2020 Nov; Vol. 61, pp. 103052. Date of Electronic Publication: 2020 Oct 09.

Advances in Pompe Disease Treatment: From Enzyme Replacement to Gene Therapy.

Autor: Colella P; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, 94305, USA. pcolella@stanford.edu.

Publikováno v: Molecular diagnosis & therapy [Mol Diagn Ther] 2024 Nov; Vol. 28 (6), pp. 703-719. Date of Electronic Publication: 2024 Aug 12.

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice.

Autor: Colella P; Genethon, INSERM U951 Integrare, University of Evry, Université Paris-Saclay, 91002, Evry, France., Sellier P; Genethon, INSERM U951 Integrare, University of Evry, Université Paris-Saclay, 91002, Evry, France.; University Pierre and Marie Curie Paris 6 and INSERM U974, 75651, Paris, France., Costa Verdera H; Genethon, INSERM U951 Integrare, University of Evry, Université Paris-Saclay, 91002, Evry, France.; University Pierre and Marie Curie Paris 6 and INSERM U974, 75651, Paris, France., Puzzo F; Genethon, INSERM U951 Integrare, University of Evry, Université Paris-Saclay, 91002, Evry, France., van Wittenberghe L; Genethon, INSERM U951 Integrare, University of Evry, Université Paris-Saclay, 91002, Evry, France., Guerchet N; Genethon, INSERM U951 Integrare, University of Evry, Université Paris-Saclay, 91002, Evry, France., Daniele N; Genethon, INSERM U951 Integrare, University of Evry, Université Paris-Saclay, 91002, Evry, France., Gjata B; Genethon, INSERM U951 Integrare, University of Evry, Université Paris-Saclay, 91002, Evry, France., Marmier S; University Pierre and Marie Curie Paris 6 and INSERM U974, 75651, Paris, France., Charles S; Genethon, INSERM U951 Integrare, University of Evry, Université Paris-Saclay, 91002, Evry, France., Simon Sola M; Genethon, INSERM U951 Integrare, University of Evry, Université Paris-Saclay, 91002, Evry, France., Ragone I; Genethon, INSERM U951 Integrare, University of Evry, Université Paris-Saclay, 91002, Evry, France., Leborgne C; Genethon, INSERM U951 Integrare, University of Evry, Université Paris-Saclay, 91002, Evry, France., Collaud F; Genethon, INSERM U951 Integrare, University of Evry, Université Paris-Saclay, 91002, Evry, France., Mingozzi F; Genethon, INSERM U951 Integrare, University of Evry, Université Paris-Saclay, 91002, Evry, France.; University Pierre and Marie Curie Paris 6 and INSERM U974, 75651, Paris, France.; Spark Therapeutics, Philadelphia, PA 19103, USA.

Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2018 Nov 17; Vol. 12, pp. 85-101. Date of Electronic Publication: 2018 Nov 17 (Print Publication: 2019).