منابع مشابه
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.
Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation factor IX (F.IX). Previously, we established an experimental basis for gene transfer as a method of treating the disease in mice and hemophilic dogs through intramuscular injection of a recombinant adeno-associated viral (rAAV) vector expressing F.IX. In this study we investigated the safety of this approach in...
متن کاملIn utero AAV-mediated gene transfer to rabbit pulmonary epithelium.
In utero intra-amniotic administration of adeno-associated virus (AAV) for treatment of cystic fibrosis (CF) has the potential to be an efficient way to target the rapidly dividing undifferentiated cells of the fetal pulmonary epithelium, while simultaneously treating other tissues involved in CF (such as the intestines), but has never before been studied. Intra-amniotic administration of 1x10(...
متن کاملImmunology of AAV-Mediated Gene Transfer in the Eye
The eye has been at the forefront of translational gene therapy largely owing to suitable disease targets, anatomic accessibility, and well-studied immunologic privilege. These advantages have fostered research culminating in several clinical trials and adeno-associated virus (AAV) has emerged as the vector of choice for many ocular therapies. Pre-clinical and clinical investigations have asses...
متن کاملTriptolide T10 enhances AAV-mediated gene transfer in mice striatum.
Adeno-associated virus (AAV) mediated gene transfer has been demonstrated to be an effective approach for treating Parkinson's disease (PD). Triptolide T10 is a monomeric compound isolated from tripterygium wilfordii Hook.f. (Thunder God vine), a traditional Chinese herb for anti-inflammatory medications. In the present study, we co-administered T10 with recombinant AAV2 in SH-SY5Y human neurob...
متن کاملFactor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.
In previous work we transferred a human factor IX-encoding adeno-associated viral vector (AAV) into skeletal muscle of men with severe hemophilia B. Biopsy of injected muscle up to 1 year after vector injection showed evidence of gene transfer by Southern blot and of protein expression by IHC and immunofluorescent staining. Although the procedure appeared safe, circulating F.IX levels remained ...
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ژورنال
عنوان ژورنال: Genetics in Medicine
سال: 2002
ISSN: 1098-3600,1530-0366
DOI: 10.1097/00125817-200211001-00012