| 摘要 |
The invention relates to the field of molecular genetics and medicine. In particular to the field of gene therapy, more in particular to gene therapy using adenoviruses. The invention provides the use of a gene delivery vehicle, more specifically an adenoviruses having tropism for stem cells, more specifically hemopoietic stem cells, wherein said tropism is provided by at least part of an adenoviral fiber protein derived from an adenovirus B serotype or functional equivalent and/or homologue thereof as a vehicle for delivering a nucleic acid to stem cells, for the treatment of Hurlers disease, Hunters disease, Sanfilippos disease, Morquois disease, Gaucher disease, Farbers disease, Niemann-pick disease, Krabbe disease, Metachromatic leucodistrophy. I-Cell disease, Fucosidose deficiency, Thallasemia and Erythropoietic porphyria, AIDS, cancer or other autoimmune diseases. |
