Zobrazeno 1 - 10
of 985
pro vyhledávání: '"AAV vectors"'
Autor:
Magalie Penaud-Budloo, Emilie Lecomte, Quentin Lecomte, Simon Pacouret, Frédéric Broucque, Aurélien Guy-Duché, Jean-Baptiste Dupont, Laurence Jeanson-Leh, Cécile Robin, Véronique Blouin, Eduard Ayuso, Oumeya Adjali
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 3, Pp 101305- (2024)
With more than 130 clinical trials and 8 approved gene therapy products, adeno-associated virus (AAV) stands as one of the most popular vehicles to deliver therapeutic DNA in vivo. One critical quality attribute analyzed in AAV batches is the presenc
Externí odkaz:
https://doaj.org/article/042640a504cc42909af1d623d410fa67
Publikováno v:
Frontiers in Audiology and Otology, Vol 2 (2024)
Greater understanding of the molecular intricacies of acquired and hereditary hearing loss has spurred considerable advances in inner ear gene therapy. While approaches like cochlear amplification and cochlear implantation offer varying degrees of ef
Externí odkaz:
https://doaj.org/article/4ab5bbe0d82c450b84c04eb2bbd74a12
Autor:
Yuan Lu, Chen Ling, Jakob Shoti, Hua Yang, Aneesha Nath, Geoffrey D. Keeler, Keyun Qing, Arun Srivastava
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 2, Pp 102196- (2024)
We identified that distal 10 nucleotides in the D-sequence in AAV2 inverted terminal repeat (ITR) share partial sequence homology to 1/2 binding site of glucocorticoid receptor-binding element (GRE). Here, we describe that (1) purified GR binds to AA
Externí odkaz:
https://doaj.org/article/6b4690e0cbbd46d6b95f99b3995ba803
Autor:
Estera Rintz, Betul Celik, Nidhi Fnu, Angélica María Herreño-Pachón, Shaukat Khan, Eliana Benincore-Flórez, Shunji Tomatsu
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 2, Pp 102211- (2024)
Mucopolysaccharidosis type IVA (MPS IVA) is caused by a deficiency of the galactosamine (N-acetyl)-6-sulfatase (GALNS) enzyme responsible for the degradation of specific glycosaminoglycans (GAGs). The progressive accumulation of GAGs leads to various
Externí odkaz:
https://doaj.org/article/2f3e9aa130624ddab430b233b94c89ae
Autor:
Ambra Cappelletto, Edoardo Alfì, Nina Volf, Thi Van Anh Vu, Francesca Bortolotti, Giulio Ciucci, Simone Vodret, Marco Fantuz, Martina Perin, Andrea Colliva, Giacomo Rozzi, Matilde Rossi, Giulia Ruozi, Lorena Zentilin, Roman Vuerich, Daniele Borin, Romano Lapasin, Silvano Piazza, Mattia Chiesa, Daniela Lorizio, Luca Triboli, Sandeep Kumar, Gaia Morello, Claudio Tripodo, Maurizio Pinamonti, Giulia Maria Piperno, Federica Benvenuti, Alessandra Rustighi, Hanjoong Jo, Stefano Piccolo, Giannino Del Sal, Alessandro Carrer, Mauro Giacca, Serena Zacchigna
Publikováno v:
Journal of Experimental & Clinical Cancer Research, Vol 43, Iss 1, Pp 1-14 (2024)
Abstract Background New drugs to tackle the next pathway or mutation fueling cancer are constantly proposed, but 97% of them are doomed to fail in clinical trials, largely because they are identified by cellular or in silico screens that cannot predi
Externí odkaz:
https://doaj.org/article/a789156b8a0b44008d091dcfb970d332
Autor:
Mariagiovanna Russo, Amina Zahaf, Abdelmoumen Kassoussi, Ariane Sharif, Hélène Faure, Elisabeth Traiffort, Martial Ruat
Publikováno v:
Cells, Vol 13, Iss 21, p 1808 (2024)
Failure of myelin regeneration by oligodendrocytes contributes to progressive decline in many neurological diseases. Here, using in vitro and in vivo rodent models, functional blockade, and mouse brain demyelination, we demonstrate that Sonic hedgeho
Externí odkaz:
https://doaj.org/article/ae38fecd7da84749adef8bb4063a6a48
Autor:
Junping Zhang, Dylan A. Frabutt, Matthew Chrzanowski, Ning Li, Lohra M. Miller, Jiahe Tian, Patrick L. Mulcrone, Anh K. Lam, Benjamin E. Draper, Martin F. Jarrold, Roland W. Herzog, Weidong Xiao
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 1, Pp 101206- (2024)
Self-complementary AAV vectors (scAAV) use a mutant inverted terminal repeat (mITR) for efficient packaging of complementary stranded DNA, enabling rapid transgene expression. However, inefficient resolution at the mITR leads to the packaging of mono
Externí odkaz:
https://doaj.org/article/ba0fb73ec32b4ab08090643c484cf170
Autor:
Martin Bentler, Romain Hardet, Moritz Ertelt, Daniela Rudolf, Dorota Kaniowska, Andreas Schneider, Florian W.R. Vondran, Clara T. Schoeder, Marion Delphin, Julie Lucifora, Michael Ott, Ulrich T. Hacker, Sahil Adriouch, Hildegard Büning
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 576-592 (2023)
De novo immune responses are considered major challenges in gene therapy. With the aim to lower innate immune responses directly in cells targeted by adeno-associated virus (AAV) vectors, we equipped the vector capsid with a peptide known to interfer
Externí odkaz:
https://doaj.org/article/653f4c03268045f68435d394aae772b1
Autor:
Arun Srivastava
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 32, Iss , Pp 949-959 (2023)
Recombinant adeno-associated virus (AAV) vectors have been, or are currently in use, in 332 phase I/II/III clinical trials in a number of human diseases, and in some cases, remarkable clinical efficacy has also been achieved. There are now three US F
Externí odkaz:
https://doaj.org/article/b9fbbd3028474b6dafdc228ac8d29e8b
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 31, Iss , Pp 101147- (2023)
The first generation of adeno-associated virus (AAV) vectors composed of the naturally occurring capsids and genomes, although effective in some instances, are unlikely to be optimal for gene therapy in humans. The use of the first generation of two
Externí odkaz:
https://doaj.org/article/ac8785c443034e108a8cac0109fe16e2