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Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease.

Retroviral gene transfer has been used successfully to correct the glucocerebrosidase (GCase) deficiency in primary hematopoietic cells from patients with Gaucher disease. For this model of somatic gene therapy, we developed a high-titer, amphotropic retroviral vector designated NTG in which the hum...

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書誌詳細
主要な著者: Fink, J K, Correll, P H, Perry, L K, Brady, R O, Karlsson, S
フォーマット: Artigo
言語:Inglês
出版事項: 1990
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC53681/
https://ncbi.nlm.nih.gov/pubmed/2315324
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