Zobrazeno 1 - 10
of 13
pro vyhledávání: '"Jose Bonafont"'
Autor:
Stuart Ellison, Karen Buckland, Yuko Learmonth, Victoria Day, Spandan Kalra, Lauren Howe, Francisco José Roman-Rodriguez, Jose Bonafont, Laura Booth, Rebecca Holley, Jon Smythe, Simon Jones, Adrian Thrasher, Claire Booth, Brian W. Bigger
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 2, Pp 101271- (2024)
Hematopoietic stem cell gene therapy (HSCGT) is a promising therapeutic strategy for the treatment of neurodegenerative, metabolic disorders. The approach involves the ex vivo introduction of a missing gene into patients’ own stem cells via lentivi
Externí odkaz:
https://doaj.org/article/c6d56cc1cb214a2393e79a6e7acc9046
Autor:
Marta García, Jose Bonafont, Jesús Martínez-Palacios, Rudan Xu, Giandomenico Turchiano, Stina Svensson, Adrian J. Thrasher, Fernando Larcher, Marcela Del Rio, Rubén Hernández-Alcoceba, Marina I. Garín, Ángeles Mencía, Rodolfo Murillas
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 96-108 (2022)
Recessive dystrophic epidermolysis bullosa, a devastating skin fragility disease characterized by recurrent skin blistering, scarring, and a high risk of developing squamous cell carcinoma is caused by mutations in COL7A1, the gene encoding type VII
Externí odkaz:
https://doaj.org/article/d7c843aebbdf4538b32d4bed782a099b
Autor:
Victoria Gálvez, Esteban Chacón-Solano, Jose Bonafont, Ángeles Mencía, Wei-Li Di, Rodolfo Murillas, Sara Llames, Asunción Vicente, Marcela Del Rio, Marta Carretero, Fernando Larcher
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 280-290 (2020)
Current efforts to find specific genodermatoses treatments and define precise pathogenesis mechanisms require appropriate surrogate models with human cells. Although transgenic and gene knockout mouse models for several of these disorders exist, they
Externí odkaz:
https://doaj.org/article/d75b071d40cb45b5b8edf78f7f767001
Autor:
Ángeles Mencía, Cristina Chamorro, Jose Bonafont, Blanca Duarte, Almudena Holguin, Nuria Illera, Sara G. Llames, Maria José Escámez, Ingrid Hausser, Marcela Del Río, Fernando Larcher, Rodolfo Murillas
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 11, Iss C, Pp 68-78 (2018)
Recessive dystrophic epidermolysis bullosa is a severe skin fragility disease caused by loss of functional type VII collagen at the dermal-epidermal junction. A frameshift mutation in exon 80 of COL7A1 gene, c.6527insC, is highly prevalent in the Spa
Externí odkaz:
https://doaj.org/article/1fa63878cdfa4572adda6f761660cb5f
Autor:
R. Foley, Jennifer Lynch, Jonathan O’Keeffe Ahern, Qian Xu, Sigen A, Jose Bonafont, Rodolfo Murillas, Marta García, Ángeles Mencía, Irene Lara-Sáez, Darío Manzanares, Wenxin Wang, Fernando Larcher, Dezhong Zhou
Publikováno v:
Gene Therapy. 29:157-170
Recent advances in molecular biology have led to the CRISPR revolution, but the lack of an efficient and safe delivery system into cells and tissues continues to hinder clinical translation of CRISPR approaches. Polymeric vectors offer an attractive
Autor:
Sara Llames, E. Chacón-Solano, Jose Bonafont, Asunción Vicente, Marcela Del Rio, Victoria Galvez, Ángeles Mencía, Fernando Larcher, Marta Carretero, Wei-Li Di, Rodolfo Murillas
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss, Pp 280-290 (2020)
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss, Pp 280-290 (2020)
Current efforts to find specific genodermatoses treatments and define precise pathogenesis mechanisms require appropriate surrogate models with human cells. Although transgenic and gene knockout mouse models for several of these disorders exist, they
Autor:
Matthew H. Porteus, Rosa Romano, Rodolfo Murillas, Wai Srifa, Jose Bonafont, Fernando Larcher, Marta García, Marcela Del Rio, Ángeles Mencía, Rosario Hervás-Salcedo, Sriram Vaidyanathan, Laura Ugalde, Blanca Duarte, E. Chacón-Solano
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy. 29(6)
Genome-editing technologies that enable the introduction of precise changes in DNA sequences have the potential to lead to a new class of treatments for genetic diseases. Epidermolysis bullosa (EB) is a group of rare genetic disorders characterized b
Autor:
Raul M. Torres, María José Escámez, Carlos León, E. Chacón-Solano, Marcela Del Rio, Lucía Marinas, Marta García, Silvia Modamio-Høybjør, Jose Bonafont, Ángeles Mencía, Sandra Rodriguez, Marta Carretero, Fernando Larcher, Ingrid Hausser, Rodolfo Murillas
Publikováno v:
Repisalud
Instituto de Salud Carlos III (ISCIII)
Molecular Therapy
e-Archivo. Repositorio Institucional de la Universidad Carlos III de Madrid
instname
Instituto de Salud Carlos III (ISCIII)
Molecular Therapy
e-Archivo. Repositorio Institucional de la Universidad Carlos III de Madrid
instname
Gene editing constitutes a novel approach for precisely correcting disease-causing gene mutations. Frameshift mutations in COL7A1 causing recessive dystrophic epidermolysis bullosa are amenable to open reading frame restoration by non-homologous end
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2f903e859eaa2881c8d991d6f99881dc
http://hdl.handle.net/20.500.12105/7611
http://hdl.handle.net/20.500.12105/7611
Autor:
José Miguel Soriano, José Cervera, Valentín Esteban, M. Almonacid, Esperanza Such, Alegría Montoro, Gumersindo Verdú, Joan Francesc Barquinero, Natividad Sebastià, Jose Bonafont, David Hervás, J. I. Villaescusa
Publikováno v:
RiuNet. Repositorio Institucional de la Universitat Politécnica de Valéncia
instname
GENETIC TESTING AND MOLECULAR BIOMARKERS
r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe
instname
GENETIC TESTING AND MOLECULAR BIOMARKERS
r-IIS La Fe. Repositorio Institucional de Producción Científica del Instituto de Investigación Sanitaria La Fe
[EN] Background: A combination of several factors including a change in the paint application system; a lack of proper hygiene; and inadequate safety measures caused a severe health impact in the workers of some textile painting factories. This outbr
Autor:
Rodolfo Murillas, Almudena Holguín, Ángeles Mencía, Fernando Larcher, Nuria Illera, C.A. Chamorro, Marcela Del Rio, Ingrid Hausser, Blanca Duarte, Jose Bonafont, Sara Llames, María José Escámez
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 11, Iss C, Pp 68-78 (2018)
Molecular Therapy. Nucleic Acids
e-Archivo. Repositorio Institucional de la Universidad Carlos III de Madrid
instname
Molecular Therapy. Nucleic Acids
e-Archivo. Repositorio Institucional de la Universidad Carlos III de Madrid
instname
Recessive dystrophic epidermolysis bullosa is a severe skin fragility disease caused by loss of functional type VII collagen at the dermal-epidermal junction. A frameshift mutation in exon 80 of COL7A1 gene, c.6527insC, is highly prevalent in the Spa
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e34ad1edeca46349ffa72dd8df36162c
https://hdl.handle.net/10016/28742
https://hdl.handle.net/10016/28742