A two-pronged approach to generating novel models of photoreceptor degeneration for regenerative cell therapy

Ended Cooperative Agreement Awarded Last action

Award ID
U24EY033272
Recipient
Awarding agency
Obligated
$10,509,662

Award facts

Assistance listing
93.867 Vision Research
Completion date
Funding
  • $9,472,115.00 Federal
  • $1,037,547.00 Non-federal
Place of performance
Houston, TX 77030, USA

Award details come from USAspending.gov.

Description

As the agency reported it.

Abstract
Inherited retinal diseases (IRD), such as retinitis pigmentosa (RP), leber congenital amaurosis (LCA), are a common cause of visual impairment worldwide. Recent studies show that cell replacement therapy is a promising therapeutic option for IRD patients with severe retinal degeneration that destroys photoreceptors. We and others recently reported long-term survival and integration of human embryonic stem cells (hescs) or human induced pluripotent stem cells (hipscs) derived retinas in a novel laser-induced photoreceptor degeneration model. While the laser ablation model can be generated rapidly as an acute degeneration model, IT does not have the progressive pathophysiological nature (e.g., disease time course, retinal remodeling, etc.) of IRD. To address this issue, we developed a highly efficient genome-editing technology, platinum talen, which leads to gene disruption in multiple organisms with high efficacy and specificity. By applying platinum talen to this novel model, we achieved one-step generation of biallelic gene modifications with high efficiency (40-50%) in preliminary studies. Since substantial information regarding the survival and integration of transplanted photoreceptor cells or retinal sheets have been obtained in laser injury models, we propose a two-pronged strategy to generating novel translation-enabling models to evaluate survival and integration of regenerated photoreceptors in the visual system: 1) to use laser-induced photoreceptor degeneration model; 2) one-step generation of biallelic mutants by platinum talen. We will then systematically compare the survival and integration of regenerated photoreceptors in the two model systems. Aim 1 will generate novel models of photoreceptor degeneration by laser ablation and platinum talen. Aim 2 will perform comprehensive ophthalmic phenotyping of laser-induced and talen-edited models of photoreceptor degeneration. Aim 3 will evaluate survival and integration of human induced pluripotent stem cell (hipsc)-derived retina sheets in the two models of photoreceptor degeneration.

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Show all 33

Subawards

Subrecipient Amount Awarded
National University Corporation, Kyoto University Agreement: $138,273 $138,273
Subrecipient not on record Agreement: $287,564 Amd 1: $283,773 Amd 2: $284,014 Amd 3: $0 (P700000165) Amd 4: $0 (P700000165) Amd 5: $183,270 (P700000165) Takeshi Iwata is responsible… $1,038,621
Yale Univ Agreement: $167,925 I confirm my commitment to serve as a co-investigator on Dr. Tran’s R01 application “Activity-related mechanisms of selective… $167,925
Kobe City Eye Hospital Agreement:$140,622 Amd1: $55,784 Amd2: $240,123 Amd3: $0 Amd 4: $263,408 Amd 5: $0 Amd 6: $239,684 Subrecipient will conduct 1 Evaluate the monkey retinal… $699,937
Hiroshima University Agreement: $142,749 Amd1: $140,325 Amd2: $0 Amd3: $139,895 Sakuma and colleagues previously developed a highly active genome editing system, Platinum TALEN,… $422,969
Riken Yr 1 $137,016 Yr 1 Amd 1: -$55,784.46 Subrecipient plans to conduct the following works (year-1) 1 Prepare the laser induced perimacular retinal degeneration… $81,232

Quick answers

Who received award U24EY033272?

U24EY033272 was awarded to Baylor College Of Medicine by National Eye Institute (National Institutes of Health) on September 30, 2021.

How much is award U24EY033272 worth?

$10,509,662 has been obligated on U24EY033272: $9,472,115 in federal funding and $1,037,547 in non-federal funding.

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