Konu "Gene therapy" için Kök Hücre Araştırma ve Uygulama Merkezi (PEDISTEM) Makale Koleksiyonu listeleme
Toplam kayıt 2, listelenen: 1-2
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Lentiviral Hematopoietic Stem Cell Gene Therapy in Inherited Immune and Lysosomal Enzyme Deficiencies
(CyberLeninka, 2016)Rare diseases affect millions of people worldwide. Many of those are inherited disorders resulting in chronic disability and requiring cost-intensive care. Hematopoietic stem cell gene therapy has been developed over more ... -
Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of Mngie
(Elsevier, 2018)Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an autosomal recessive disorder caused by thymidine phosphorylase (TP) deficiency resulting in systemic accumulation of thymidine (d-Thd) and deoxyuridine ...
