A dual-vector system was explored for the delivery of the coagulation factor VIII gene, using intein-mediated protein trans-splicing as a means to produce intact functional factor VIII post-translationally.

Cancer/testis (CT) antigens exhibit highly tissue-restricted expression and are considered promising targets for cancer vaccines.

Congenital erythropoietic porphyria (CEP) is a severe autosomal recessive disorder characterized by a deficiency in uroporphyrinogen III synthase (UROS), the fourth enzyme of the heme biosynthetic pathway.

* In phase 2 metastatic melanoma study of 50 patients with stage IIIC and IV disease, 10 achieved complete response

* Planned phase 3 study of head and neck SCC will involve 400 patients with advanced nodal disease who will be randomized to standard therapy with radiation plus cisplatin or combination with OncoVEX.

The N-termini of capsid proteins VP1 and VP2 in adeno-associated virus (AAV) play important roles in subcellular steps of infection and contain motifs that are highly homologous to a phospholipase A2 (PLA2) domain and nuclear localization signals (NLSs).

Cincinnati Children's Hospital Medical Center has gotten permission from the U.S. Food and Drug Administration to test a gene therapy treatment against X-linked severe combined immunodeficiency, or so-called "bubble-boy" disease. It's the first federal approval the Corryville hospital has received for a gene therapy treatment conceived and manufactured in its own lab.

AGTC, a privately held company, and Icagen, Inc. /quotes/comstock/15*!icgn/quotes/nls/icgn (ICGN 0.44, 0.00, 0.00%) today announced the completion of an agreement for the purchase and sale of certain patent rights between the companies. Icagen is transferring its rights to patents relating to the ion channel gene CNGB3, which has been linked to certain disorders of the eye.

Poor gene transfer efficiency has been a major problem in developing an effective gene therapy for cystic fibrosis (CF) airway disease. Lysophosphatidylcholine (LPC), a natural airway surfactant, can enhance viral gene transfer in animal models.

“A single treatment cured about 50 percent of the diabetic mice, restoring their blood sugar to normal so that they no longer need insulin injections,” said study co-author Lawrence Chan, chief of Baylor’s diabetes, endocrinology and metabolism division.

Neurologix, Inc. (OTC Bulletin Board: NRGX), today announced positive results in a Phase 2 trial of its investigational gene therapy for advanced Parkinson's disease (PD), NLX-P101. Study participants who received NLX-P101 experienced statistically significant and clinically meaningful improvements in off-medication motor scores compared to control subjects who received sham surgery.

STEMCELL Technologies Inc announced today that they have signed a licensing agreement with Stanford University for a virus-free technique used for the generation of induced pluripotent stem cells. This technology will soon be commercially released by STEMCELL Technologies as STEMcircles™.