Making the (clinical) grade with adenoviral gene therapy vectors

Adenoviral Vectors for Hemophilia Gene Therapy

Hemophilia is an inherited blood clotting disorder resulting from deficiency of blood coagulation factors. Current standard of care for hemophilia patients is frequent intravenous infusions of the missing coagulation factor. Gene therapy for hemophilia involves the introduction of a normal copy of the deficient coagulation factor gene thereby potentially offering a definitive cure for the bleed...

Gene therapy of Osteosarcoma Preclinical studies with adenoviral vectors and adenoviral oncolysis

Despite improvement in the treatment of osteosarcoma there are still many patients who cannot benefit from current treatment modalities. This warrants exploration of new treatment options. To that end, we investigated Gene-Directed Enzyme Prodrug Therapy (GDEPT) with the use of human liver carboxylesterase-2 (CE2) and the anticancer agent CPT-11. CPT-11 is a clinically approved prodrug that nee...

Gene Therapy of Glioblastoma Multiforme - Clinical Experience on the Use of Adenoviral Vectors

Helper-dependent adenoviral vectors in experimental gene therapy.

In the majority of potential applications gene therapy will require an effective transfer of a transgene in vivo resulting in high-level and long-term transgene expression, all in the absence of significant toxicity or inflammatory responses. The most efficient vehicles for delivery of foreign genes to the target tissues are modified adenoviruses. Adenoviral vectors of the first generation, des...

Targeting adenoviral vectors for enhanced gene therapy of uterine leiomyomas.

STUDY QUESTION Is targeted adenovirus vector, Ad-SSTR-RGD-TK (Adenovirus -human somatostatin receptor subtype 2- arginine, glycine and aspartate-thymidine kinase), given in combination with ganciclovir (GCV) against immortalized human leiomyoma cells (HuLM) a potential therapy for uterine fibroids? SUMMARY ANSWER Ad-SSTR-RGD-TK/GCV, a targeted adenovirus, effectively reduces cell growth in Hu...