Gene-based agents for the treatment of congenital eye diseases

ByConnor Hodde

Pharmacologists at LMU have developed gene-based agents for the treatment of congenital eye diseases. The first of these is now undergoing a phase-I clinical trial in color-blind patients at the University Medical Center in Tübingen.

Is this approach translatable in principle to other visual disorders?

Michalakis: About 200 genes have been identified which, when mutated, lead to monogenetic visual diseases, and doubtless many more remain to be discovered. Defects in any of 50 different genes can result in retinitis pigmentosa, a degenerative disease in which specific retinal photoreceptors die. Six genes have been linked to achromatopsia, and all of them should be amenable to the approach we are using to correct for defects in the CNGA3 gene.

Read the entire article here.

Similar Posts

Interim results from clinical trial demonstrate safety of cell-based therapy for retinitis pigmentosa

ByConnor Hodde

Regenerative medicine company jCyte and the Sue & Bill Gross Stem Cell Research Center at the University of California, Irvine report that their investigational therapy for retinitis pigmentosa (RP) has demonstrated a favorable safety and tolerability profile in an ongoing Phase I/II clinical trial.  The cell-based approach taken is intended to rescue sick and dying…

Is Treat-and-Extend Truly Effective for Diabetic Macular Edema?

ByConnor Hodde

“Anti-vascular endothelial growth factor (anti-VEGF) medications have a proven track record in treating diabetic macular edema (DME). However, researchers continue to investigate whether these medications can be made more efficient and cost-effective.” “Both the RETAIN[1] and REACT[2] studies showed that treat-and-extend regimens for ranibizumab were efficacious for DME. Further support was provided by the TREX-DME study, the first prospective, randomized controlled…

SDM Photocoagulation Superior to Intravitreal Injections of BCZ in the Treatment of CSC

ByConnor Hodde

A recent study was conducted to evaluate the treatment of central serous chorioretinopathy (CSC) with either subthreshold diode laser MicroPulse (SDM) or intravitreal bevacizumab (BCZ). The study was conducted over a period of 10 months and examined 52 eyes of 52 patients with (a) treatment with SDM at the active leakage site guided by fluorescein…

Adolescents with Diabetes Experiencing Decrease in Retinopathy

ByConnor Hodde

A long-term study from Australia has claimed that there has been a significant reduction in the number of cases of retinopathy among young patients with type 1 diabetes who have had intensive glycaemic control therapy. http://www.diabetes.co.uk/news/2011/Oct/adolescents-with-diabetes-experiencing-decrease-in-retinopathy-95087803.html

Optic Nerve Regeneration in Mice Linked to Improved Vision

ByConnor Hodde

Interventions resulting in optic nerve regeneration restored some components of vision, according to the results of a mouse model study published online May 21 issue of the Proceedings of the National Academy of Sciences. If these findings are confirmed and extended to other models, they may ultimately offer promise to patients with glaucoma or optic…