Advanced Cell Technology (ACT), based in Marlborough, Mass., will begin testing
its retinal cell treatment this year in a dozen patients with Stargardt's macular dystrophy, an inherited degenerative eye disease that leads to blindness in children.
Not exact matches
Similar patches and
treatments have already been tested against the «wet» form — in which blood vessels invade and destroy the
retinal pigment epithelial
cells that nourish and support the photoreceptor
cells that capture light.
A new gene therapy
treatment has restored some sight in a handful of blind patients suffering from Leber's congenital amaurosis, a syndrome in which, because of a broken or missing gene called RPE65,
retinal photoreceptor
cells malfunction and eventually die.
Spark CEO Jeff Marrazzo says their more potent vector and differences in how the
treatment is made, such as the company's use of a surfactant to make sure the vector doesn't stick to the vial when the surgeon injects it, may result in a higher dose of the RPE65 gene getting into
retinal cells and long - lasting effects.
The research team's next step will be to evaluate how the Amyloid beta proteins get into
retinal cells and study more closely how damage occurs, with a view of establishing preventative measures or
treatment options.
A trial in 18 people with damaged
retinal cells is being hailed as the most promising yet for a
treatment based on human embryonic stem
cells (hESCs).
«Current
treatments can slow these diseases» progression, but they can't replace lost
retinal cells.
Dominic Eberle (Ader, TUD)-- «
Cell transplantation and gene therapy approaches for the
treatment of
retinal degenerative disorders» (2012)
However, current results point to a possible role for hNPCctx in the
treatment of at least some forms of human
retinal degenerative diseases and highlight the versatility and efficacy of these
cells as therapeutic tools in a broad range of neurodegenerative disorders [10]--[12], [66].
Many of the most promising approaches fall into four categories: the
retinal prosthetic, gene therapy, stem
cell treatments, and a technique that uses optogenetics, a way to engineer nerves to fire in response to bursts of light.
The
treatment could help people who have a fault in a gene called RPE65, which causes problems in the
retinal pigment epithelium (RPE), a thin layer of
cells that support and nourish photoreceptors.
A non-nucleic acid based reprogramming strategy promises to provide a safe and effective
treatment for lost
retinal cells in glaucoma sufferers.
The stem
cell approach I am most excited about involves a
treatment to replace
cells called
retinal pigment epithelial
cells.
The successful differentiation of iPS
cells into RPE represents a significant advance in the search for a potential
cell source for the
treatment of human neural
retinal diseases.
(2007) Chronic Minocycline
Treatment Decreases Microglial Activation and Improves Retrograde Axonal Transport in
Retinal Ganglion
Cells in DBA / 2J Retina (Investigative Ophthalmology & Visual Science, 48: ARVO E-Abstract 3291).
Empowered with the knowledge that
retinal cells play a critical role in communicating information from the eye to brain, researchers have begun to develop a suite of potential
treatments and therapies.