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Article

Development of lentiviral vectors with optimized transcriptional activity for the gene therapy of patients with Fanconi anemia.

Human gene therapy - 1 May 2010

González-Murillo Africa, Lozano M Luz, Alvarez Lara, Jacome Ariana, Almarza Elena, Navarro Susana, Segovia José C, Hanenberg Helmut, Guenechea Guillermo, Bueren Juan A, Río Paula

Abstract excerpt

Fanconi anemia (FA) is an inherited genetic disease characterized mainly by bone marrow failure and cancer predisposition. Although gene therapy may constitute a good therapeutic option for many patients with FA, none of the clinical trials so far developed has improved the clinical status of these patients. We have proposed strategies for the genetic correction of bone marrow grafts from patients with FA, using...

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PMID: 20001454
DOI: 10.1089/hum.2009.141

Genes

FANCA

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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