Zobrazeno 1 - 10
of 84
pro vyhledávání: '"Gloria Gonzalez-Aseguinolaza"'
Autor:
Gracián Camps, Sheila Maestro, Laura Torella, Diego Herrero, Carla Usai, Martin Bilbao-Arribas, Ana Aldaz, Cristina Olagüe, Africa Vales, Lester Suárez-Amarán, Rafael Aldabe, Gloria Gonzalez-Aseguinolaza
Publikováno v:
PLoS Pathogens, Vol 20, Iss 5, p e1011749 (2024)
Hepatitis delta virus (HDV) infection represents the most severe form of human viral hepatitis; however, the mechanisms underlying its pathology remain incompletely understood. We recently developed an HDV mouse model by injecting adeno-associated vi
Externí odkaz:
https://doaj.org/article/f65b905aaaa044dfbfa7b73578b995c9
Autor:
Sheila Maestro, Nahia Gomez-Echarte, Gracian Camps, Carla Usai, Cristina Olagüe, Africa Vales, Rafael Aldabe, Gloria Gonzalez-Aseguinolaza
Publikováno v:
Viruses, Vol 16, Iss 3, p 379 (2024)
Hepatitis D virus (HDV) infection represents the most severe form of chronic viral hepatitis. We have shown that the delivery of HDV replication-competent genomes to the hepatocytes using adeno-associated virus (AAV-HDV) as gene delivery vehicles off
Externí odkaz:
https://doaj.org/article/a9720236fa12448a89ef2f6eeb74da02
Autor:
Oihana Murillo, Maria Collantes, Cristina Gazquez, Daniel Moreno, Ruben Hernandez-Alcoceba, Miren Barberia, Margarita Ecay, Blanche Tamarit, Anne Douar, Veronica Ferrer, Jean Philippe Combal, Ivan Peñuelas, Bernard Bénichou, Gloria Gonzalez-Aseguinolaza
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 26, Iss , Pp 98-106 (2022)
Wilson’s disease (WD) is an inherited disorder of copper metabolism associated with mutations in ATP7B gene. We have shown that the administration of an adeno-associated vector (AAV) encoding a mini version of human ATP7B (VTX-801) provides long-te
Externí odkaz:
https://doaj.org/article/afe87d2e90484e9ca008bbd452940d81
Autor:
Alejandro Fernandez-Montero, Jon Zuaznabar, Manuel Pina-Sanchez, Sheila Maestro, Loreto Martin-Navarro, Natalia Muñoz-Rodríguez, Cristina Olagüe, Marta Pastrana, Maria Martínez-Fernández, Gracian Camps, Jose Antonio Rodriguez, Francesco P. Marchese, Jon Zazpe, Marta Pozuelo, José Luis Del Pozo, Jorge Quiroga, Antonio Pineda-Lucena, Gabriel Reina, Jack Kolenda, Laura Moreno-Galarraga, Gloria Gonzalez-Aseguinolaza, Marta Rua, Cristian Smerdou, Francisco Carmona-Torre, Josepmaria Argemi
Publikováno v:
Frontiers in Cellular and Infection Microbiology, Vol 13 (2023)
BackgroundThe main objective was to evaluate the efficacy of intranasal photodynamic therapy (PDT) in SARS-CoV-2 mildly symptomatic carriers on decreasing the infectivity period. SARS-CoV-2-specific immune-stimulating effects and safety were also ana
Externí odkaz:
https://doaj.org/article/6218370b66db4d6188bcb40fa06dd4fd
Autor:
Sara Lumbreras, Ana Ricobaraza, Lucia Baila-Rueda, Manuela Gonzalez-Aparicio, Lucia Mora-Jimenez, Iker Uriarte, Maria Bunuales, Matias A. Avila, Maria J. Monte, Jose J.G. Marin, Ana Cenarro, Gloria Gonzalez-Aseguinolaza, Ruben Hernandez-Alcoceba
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 22, Iss , Pp 210-221 (2021)
Cerebrotendinous xanthomatosis (CTX) is an autosomal recessive disease caused by mutations in the CYP27A1 gene, encoding the sterol 27-hydroxylase. Disruption of the bile acid biosynthesis pathway and accumulation of toxic precursors such as cholesta
Externí odkaz:
https://doaj.org/article/dbca431b41004c1fa3d3a6751b4526b2
Autor:
Lucia Mora-Jimenez, Miguel Valencia, Rocio Sanchez-Carpintero, Jan Tønnesen, Saja Fadila, Moran Rubinstein, Manuela Gonzalez-Aparicio, Maria Bunuales, Eva Fernandez-Pierola, Maria Jesus Nicolas, Elena Puerta, Cristina Miguelez, Paula Gimenez Minguez, Sara Lumbreras, Gloria Gonzalez-Aseguinolaza, Ana Ricobaraza, Ruben Hernandez-Alcoceba
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 25, Iss , Pp 585-602 (2021)
Dravet syndrome is a genetic encephalopathy characterized by severe epilepsy combined with motor, cognitive, and behavioral abnormalities. Current antiepileptic drugs achieve only partial control of seizures and provide little benefit on the patient
Externí odkaz:
https://doaj.org/article/d8a21f6b96294c4686222a2f4ad8317e
Autor:
Moanaro Biswas, Damien Marsic, Ning Li, Chenhui Zou, Gloria Gonzalez-Aseguinolaza, Irene Zolotukhin, Sandeep R.P. Kumar, Jyoti Rana, John S.S. Butterfield, Oleksandr Kondratov, Ype P. de Jong, Roland W. Herzog, Sergei Zolotukhin
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 19, Iss , Pp 347-361 (2020)
Limitations to successful gene therapy with adeno-associated virus (AAV) can comprise pre-existing neutralizing antibodies to the vector capsid that can block cellular entry, or inefficient transduction of target cells that can lead to sub-optimal ex
Externí odkaz:
https://doaj.org/article/05f6a412488548bf8d9ee4437f4100f3
Autor:
Andrea Bazo, Aquilino Lantero, Itsaso Mauleón, Leire Neri, Martin Poms, Johannes Häberle, Ana Ricobaraza, Bernard Bénichou, Jean-Philippe Combal, Gloria Gonzalez-Aseguinolaza, Rafael Aldabe
Publikováno v:
International Journal of Molecular Sciences, Vol 23, Iss 23, p 14940 (2022)
Citrullinemia type I (CTLN1) is a rare autosomal recessive disorder caused by mutations in the gene encoding argininosuccinate synthetase 1 (ASS1) that catalyzes the third step of the urea cycle. CTLN1 patients suffer from impaired elimination of nit
Externí odkaz:
https://doaj.org/article/3c5c85fe61534425b1179f700c11b6e0
Autor:
Sheila Maestro, Nicholas D. Weber, Nerea Zabaleta, Rafael Aldabe, Gloria Gonzalez-Aseguinolaza
Publikováno v:
JHEP Reports, Vol 3, Iss 4, Pp 100300- (2021)
Summary: Gene therapy is becoming an increasingly valuable tool to treat many genetic diseases with no or limited treatment options. This is the case for hundreds of monogenic metabolic disorders of hepatic origin, for which liver transplantation rem
Externí odkaz:
https://doaj.org/article/bd0df39ceb9c471a9993bcdc4ec7a78b
Autor:
Nerea Zabaleta, Miren Barberia, Cristina Martin-Higueras, Natalia Zapata-Linares, Isabel Betancor, Saray Rodriguez, Rebeca Martinez-Turrillas, Laura Torella, Africa Vales, Cristina Olagüe, Amaia Vilas-Zornoza, Laura Castro-Labrador, David Lara-Astiaso, Felipe Prosper, Eduardo Salido, Gloria Gonzalez-Aseguinolaza, Juan R. Rodriguez-Madoz
Publikováno v:
Nature Communications, Vol 9, Iss 1, Pp 1-9 (2018)
Substrate reduction therapies (SRT) are a promising therapeutic approach for monogenic inherited metabolic diseases. Here the authors evaluate the therapeutic potential of an in vivo CRISPR/Cas9-mediated SRT to treat primary hyperoxaluria type I and
Externí odkaz:
https://doaj.org/article/4b179c1567b1437ea464d69ab6bf5455