Advanced Characterization of DNA Molecules in rAAV Vector Preparations by Single-stranded Virus Next-generation Sequencing

Autor: Emilie Lecomte, Benoît Tournaire, Benjamin Cogné, Jean-Baptiste Dupont, Pierre Lindenbaum, Mélanie Martin-Fontaine, Frédéric Broucque, Cécile Robin, Matthias Hebben, Otto-Wilhelm Merten, Véronique Blouin, Achille François, Richard Redon, Philippe Moullier, Adrien Léger

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 4, Iss C (2015)

Recent successful clinical trials with recombinant adeno-associated viral vectors (rAAVs) have led to a renewed interest in gene therapy. However, despite extensive developments to improve vector-manufacturing processes, undesirable DNA contaminants

Externí odkaz: https://doaj.org/article/741f46340a074fda945084019638521d

Accurate Identification and Quantification of DNA Species by Next-Generation Sequencing in Adeno-Associated Viral Vectors Produced in Insect Cells

Autor: Benjamin Cogné, Adrien Leger, Benoît Tournaire, Sylvie Saleun, Magalie Penaud-Budloo, Alain Roulet, Philippe Moullier, Pierre Lindenbaum, Eduard Ayuso, Emilie Lecomte, Véronique Blouin, Aurélien Guy-Duché, Céline Lopez-Roques

Publikováno v: Human gene therapy methods
Human gene therapy methods, New Rochelle, NY : Mary Ann Liebert, 2017, 28 (3), pp.148-162. ⟨10.1089/hgtb.2016.185⟩
Human gene therapy methods, New Rochelle, NY : Mary Ann Liebert, 2017, 28 (3), pp.148-162. 〈10.1089/hgtb.2016.185〉
Human gene therapy methods, 2017, 28 (3), pp.148-162. ⟨10.1089/hgtb.2016.185⟩

Recombinant adeno-associated viral (rAAV) vectors have proven excellent tools for the treatment of many genetic diseases and other complex diseases. However, the illegitimate encapsidation of DNA contaminants within viral particles constitutes a majo

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1ff4a427e76c6bdb647315c2f274aa23
https://pubmed.ncbi.nlm.nih.gov/28463571

Short-lived recombinant adeno-associated virus transgene expression in dystrophic muscle is associated with oxidative damage to transgene mRNA

Autor: Jean-Baptiste Dupont, Benoit Tournaire, Christophe Georger, Béatrice Marolleau, Laurence Jeanson-Leh, Mireille Ledevin, Pierre Lindenbaum, Emilie Lecomte, Benjamin Cogné, Laurence Dubreil, Thibaut Larcher, Bernard Gjata, Laetitia Van Wittenberghe, Caroline Le Guiner, Magalie Penaud-Budloo, Richard O Snyder, Philippe Moullier, Adrien Léger

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 2, Iss , Pp - (2015)

Preclinical gene therapy strategies using recombinant adeno-associated virus (AAV) vectors in animal models of Duchenne muscular dystrophy have shown dramatic phenotype improvements, but long-lasting efficacy remains questionable. It is believed that

Externí odkaz: https://doaj.org/article/846a902b7ddb4553bb393762ce9f0d6f