Zobrazeno 1 - 7
of 7
pro vyhledávání: '"Miguel Calero-Garcia"'
Autor:
Jocelyn Loo, Ian Sicher, Ailin Goff, Ockchul Kim, Nicole Clary, Alexander Alexeev, Todd Sulchek, Alla Zamarayeva, Sewoon Han, Miguel Calero-Garcia
Publikováno v:
Scientific Reports, Vol 11, Iss 1, Pp 1-11 (2021)
Abstract Messenger RNA (mRNA) delivery provides gene therapy with the potential to achieve transient therapeutic efficacy without risk of insertional mutagenesis. Amongst other applications, mRNA can be employed as a platform to deliver gene editing
Externí odkaz:
https://doaj.org/article/3c1817175ef14e56ba195de35e1cb00e
Autor:
Ian Sicher, Ockchul Kim, Alexander Alexeev, Nicole Clary, Sewoon Han, Miguel Calero-Garcia, Ailin Goff, Alla M. Zamarayeva, Todd Sulchek, Jocelyn Loo
Publikováno v:
Scientific Reports, Vol 11, Iss 1, Pp 1-11 (2021)
Scientific Reports
Scientific Reports
Messenger RNA (mRNA) delivery provides gene therapy with the potential to achieve transient therapeutic efficacy without risk of insertional mutagenesis. Amongst other applications, mRNA can be employed as a platform to deliver gene editing molecules
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5a74f76b81a76083ee824d0c6f29965d
https://doaj.org/article/3c1817175ef14e56ba195de35e1cb00e
https://doaj.org/article/3c1817175ef14e56ba195de35e1cb00e
Autor:
Ida Ricciardelli, Timothy M. Clay, Steven J. Howe, Alastair Morrison, Takis Athanasopoulos, Laurent Jespers, David J. Dow, Aurore Saudemont, Marie L. Davies, Miguel Calero-Garcia, Laure Rittié
Publikováno v:
Mol Ther
The field of cell and gene therapy (GT) is expanding rapidly and there is undoubtedly a wave of enthusiasm and anticipation for what these treatments could achieve next. Here we assessed the worldwide landscape of GT assets currently in early clinica
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::40ea5206348a6b933160ca405b99d892
https://europepmc.org/articles/PMC6822232/
https://europepmc.org/articles/PMC6822232/
Autor:
Sujal, Ghosh, Marlene, Carmo, Miguel, Calero-Garcia, Ida, Ricciardelli, Juan Carlos, Bustamante Ogando, Michael P, Blundell, Axel, Schambach, Philip G, Ashton-Rickardt, Claire, Booth, Stephan, Ehl, Kai, Lehmberg, Adrian J, Thrasher, H Bobby, Gaspar
Publikováno v:
The Journal of Allergy and Clinical Immunology
Background Mutations in the perforin 1 (PRF1) gene account for up to 58% of familial hemophagocytic lymphohistiocytosis syndromes. The resulting defects in effector cell cytotoxicity lead to hypercytokinemia and hyperactivation with inflammation in v
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=pmid________::14690577636e3f6a19b988c293ec89cf
https://pubmed.ncbi.nlm.nih.gov/29355678
https://pubmed.ncbi.nlm.nih.gov/29355678
Autor:
Miguel Calero-Garcia, H. Bobby Gaspar
Publikováno v:
Current Pediatrics Reports. 3:11-21
Severe combined immunodeficiencies (SCIDs) are a group of rare genetic diseases characterized by profound abnormalities of cellular and humoral immunity. They have been identified as ideal candidates for treatment by gene therapy and have led the dev
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::f03c46d592ade6dc0bf55a305197ebce
https://doi.org/10.1007/s40124-014-0069-1
https://doi.org/10.1007/s40124-014-0069-1
Publikováno v:
Molecular Therapy. 24:S57
Adenosine deaminase (ADA) deficiency is a rare genetic disease characterised by the absence of the ADA enzyme (EC 3.5.4.4) involved in purine salvage and degradation. Patients lacking ADA build up adenosine and deoxyadenosine metabolites in the organ
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::879009228d27f4b4912e0a2a5f8f818d
https://doi.org/10.1016/s1525-0016(16)32949-5
https://doi.org/10.1016/s1525-0016(16)32949-5
Autor:
Miguel Calero-Garcia, H. Bobby Gaspar
Publikováno v:
Cell Stem Cell. 15:529-530
CRISPR/Cas9 has recently been introduced as a gene editing tool and shows considerable promise. In this issue of Cell Stem Cell, Mandal et al. (2014) show efficient CRISPR/Cas9-mediated ablation of the CCR5 and B2M genes in primary human hematopoieti
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::42488f9eb2c390a3b512b9eacd6db18b
https://doi.org/10.1016/j.stem.2014.10.014
https://doi.org/10.1016/j.stem.2014.10.014