A new treatment for leber congenital amaurosis (LCA), a condition linked to a mutated form of RPGRIP1 that causes degeneration of photoreceptors in the eye, has proven a success in early clinical trials.
Kamolika Roy, Linda Stein and Shalesh Kaushal, from the University of Massachusetts Medical School (Worcester) have reviewed the use of recombinant adeno-associated virus (AAV) vectors for gene therapy to treat ocular diseases and published the findings in the latest issue of Human Gene Therapy.
In the study, the authors say that the approach appears to be a "safe, effective and long-term treatment for LCA", which was previously considered an untreatable disorder.
Commenting on the findings, Dr James Wilson, editor-in-chief of Human Gene Therapy, said: "The successful correction of this photoreceptor defect in a relevant mouse model of LCA should usher in a new wave of translational research in retinal degeneration syndromes."
The research complements work carried out into LCA by specialists at the Harvard Medical School, who noted that the established link between specific genetic defects and ocular disorders offers hope for using gene therapy to provide long-term benefits for sufferers.
by Martin Burns