Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal

Autor: Sara Marcó, Virginia Haurigot, Maria Luisa Jaén, Albert Ribera, Víctor Sánchez, Maria Molas, Miguel Garcia, Xavier León, Carles Roca, Xavier Sánchez, Joan Bertolin, Jennifer Pérez, Gemma Elias, Marc Navarro, Ana Carretero, Martí Pumarola, Anna Andaluz, Yvonne Espada, Sonia Añor, Fatima Bosch

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 23, Iss , Pp 370-389 (2021)

Delivery of adeno-associated viral vectors (AAVs) to cerebrospinal fluid (CSF) has emerged as a promising approach to achieve widespread transduction of the central nervous system (CNS) and peripheral nervous system (PNS), with direct applicability t

Externí odkaz: https://doaj.org/article/6c76bc2619ae4a508cc75183a3090a01

Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal

Autor: Virginia Haurigot, Xavier Sanchez, Miguel Garcia, Gemma Elias, Martí Pumarola, Anna Andaluz, Ana Carretero, Xavier León, Carles Roca, Fatima Bosch, Maria Luisa Jaén, Jennifer Pérez, Maria Molas, Victor Sanchez, Joan Bertolin, Sara Marcó, Yvonne Espada, Sònia Añor, Albert Ribera, Marc Navarro

Publikováno v: Dipòsit Digital de Documents de la UAB
Universitat Autònoma de Barcelona
Molecular Therapy: Methods & Clinical Development, Vol 23, Iss, Pp 370-389 (2021)
Molecular Therapy. Methods & Clinical Development

Delivery of adeno-associated viral vectors (AAVs) to cerebrospinal fluid (CSF) has emerged as a promising approach to achieve widespread transduction of the central nervous system (CNS) and peripheral nervous system (PNS), with direct applicability t

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8984b8c060fd37f381f7723d0eedd7ae
https://doi.org/10.1016/j.omtm.2021.09.017

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

Autor: Jesús Ruberte, Luca Maggioni, Albert Ribera, Sònia Añor, Eduard Ayuso, Maria Molas, Sara Marcó, Martí Pumarola, Virginia Haurigot, Fatima Bosch, Anna Andaluz, Federico Mingozzi, Albert Ruzo, Mercedes Pineda, Miguel Garcia, Sergio Muñoz, Sandra Motas, Pilar Villacampa, Gemma García-Fructuoso

Publikováno v: Dipòsit Digital de Documents de la UAB
Universitat Autònoma de Barcelona

For most lysosomal storage diseases (LSDs) affecting the CNS, there is currently no cure. The BBB, which limits the bioavailability of drugs administered systemically, and the short half-life of lysosomal enzymes, hamper the development of effective

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1bbcdb7ecc71012ec56147d5fda83dae
https://doi.org/10.1172/jci66778

ALOX5AP Overexpression in Adipose Tissue Leads to LXA4 Production and Protection Against Diet-Induced Obesity and Insulin Resistance

Autor: Miquel Garcia, Carles Roca, Laia Vilà, Maria Molas, Sylvie Franckhauser, Judith Agudo, Jesús Ruberte, Tura Ferre, Fatima Bosch, Alba Casellas, Sergio Muñoz, Ivet Elias

Publikováno v: Diabetes. 65(8)

Eicosanoids, such as leukotriene B4 (LTB4) and lipoxin A4 (LXA4), may play a key role during obesity. While LTB4 is involved in adipose tissue inflammation and insulin resistance, LXA4 may exert anti-inflammatory effects and alleviate hepatic steatos

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::559aeea22f16f25d86323b8e794e9bb2
https://pubmed.ncbi.nlm.nih.gov/27207555

348. Correction of CNS and Somatic Pathology by Intra-Cerebrospinal Fluid Gene Therapy for Mucopolysaccharidosis Type II

Autor: Xavier Sanchez, Carles Roca, Victor Sanchez, Jesús Ruberte, Sara Marcó, Albert Ribera, Xavier León, Maria Molas, Virginia Haurigot, Sandra Motas, Fatima Bosch, Miquel Garcia

Publikováno v: Molecular Therapy. 24:S139

Mucopolysaccharidosis type II (MPSII), or Hunter Syndrome, is a X-linked recessive Lysosomal Storage Disease (LSD) caused by deficiency in Iduronate-2-sulfatase (IDS), an enzyme involved in the stepwise degradation of the glycosaminoglycans (GAGs) he

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::38a27910d3538f26a480c126603eceb0
https://doi.org/10.1016/s1525-0016(16)33157-4