NIH re­searchers re­port an­i­mal mod­el val­i­da­tion for stem cell-based ap­proach to ad­vanced dry AMD

A group of NIH re­searchers say they have laid the ground­work need­ed to tack­le the root cause of an ad­vanced form of age-re­lat­ed mac­u­lar de­gen­er­a­tion by re­pro­gram­ming adult blood cells in­to reti­nal cells in rat and pig mod­els. And now they plan to move that in­to an his­toric break­through ef­fort in hu­mans.

The sci­en­tists from the NIH’s Na­tion­al Eye In­sti­tute set out to pre­vent blind­ness in ge­o­graph­ic at­ro­phy, or ad­vanced dry AMD. Reti­nal pig­ment ep­ithe­lial cells nur­ture pho­tore­cep­tors; when they die (usu­al­ly in the ear­ly stage of ge­o­graph­ic at­ro­phy) the pho­tore­cep­tors lose pro­tec­tion and even­tu­al­ly die as well, ul­ti­mate­ly re­sult­ing in blind­ness. With the re­sults and pro­to­cols de­tailed in a Sci­ence Trans­la­tion­al Med­i­cine pa­per to­day, they are hop­ing to set the stage for a first-in-hu­man study.

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