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Article

Rescue of pyruvate kinase deficiency in mice by gene therapy using the human isoenzyme.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Dec 2009

Meza Nestor W, Alonso-Ferrero Maria E, Navarro Susana, Quintana-Bustamante Oscar, Valeri Antonio, Garcia-Gomez Maria, Bueren Juan A, Bautista Jose M, Segovia Jose C

Abstract excerpt

Human erythrocyte R-type pyruvate kinase deficiency (PKD) is a disorder caused by mutations in the PKLR gene that produces chronic nonspherocytic hemolytic anemia. Besides periodic blood transfusion and splenectomy, severe cases require bone marrow (BM) transplant, which makes this disease a good candidate for gene therapy. Here, the normal human R-type pyruvate kinase (hRPK) complementary (cDNA) was expressed in...

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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 19755962
DOI: 10.1038/mt.2009.200
PMCID: PMC2814388

Genes

PKLR

Provenance

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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