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Setting the Stage for Replacement of Mitochondrial Genes

Award Number: R01EY010804
ORGANIZATION: NATIONAL EYE INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

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Setting the Stage for Replacement of Mitochondrial Genes - Project Summary We propose to develop a monomeric DNA editing platform to eliminated mutant mtDNA (mitoARCUS). In collaboration with Precision Biosciences, we will develop and characterize a new class of mitochondrial editing enzymes that are smaller and simpler than TALENs, while still highly specific for the mutant mtDNA. This platform is based on the I-CreI homing endonuclease, which was modified to work as a monomer, becoming more amenable to viral packaging and delivery in a clinical setting. We plan to characterize mitoArcus for three pathogenic mtDNA mutations, including: a) Human 4.9kbp Common deletion; b) Human MELAS tRNALeu(UUR) m.3243G, d) the mouse tRNAAla m.5024T. Once the mitoArcus enzymes are characterized in established cell lines, we will test their ability to change mtDNA heteroplasmy and restore function to clinically-relevant models. These will include human motor neuron organoids ex vivo and and the mouse retina in vivo. Finally, we will test a newly described base editing tool (DdCBE) in an in vivo model. This new base editor architecture (DdCBE), specifically edit thymidines on mtDNA. This was possible, because this architecture is not based on the CRISPR, thereby not requiring a gRNA component that cannot be easily imported into mitochondria. In collaboration with David Liu's lab at Harvard/Broad, we will test whether mtDNA base editing works in a mouse model harboring a pathogenic mtDNA mutation in the tRNAAla gene.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2024 ( Subtotal = $403,232 )
2024	2024	UNIVERSITY OF MIAMI	1320 S DIXIE HWY STE 1200	CORAL GABLES	FL	33146	MIAMI-DADE	USA	Vision Research	000	29	8/2/2024	NON-COMPETING CONTINUATION	$403,232
														Subtotal = $403,232

Issue Date FY: 2023 ( Subtotal = $403,232 )
2023	2023	UNIVERSITY OF MIAMI	1400 NW 10TH AVE	MIAMI	FL	33136	MIAMI-DADE	USA	Vision Research	000	28	7/20/2023	NON-COMPETING CONTINUATION	$403,232
														Subtotal = $403,232

Issue Date FY: 2022 ( Subtotal = $391,136 )
2022	2022	UNIVERSITY OF MIAMI	1600 NW 10 AVE	MIAMI	FL	33136	MIAMI-DADE	USA	Vision Research	002	27	9/8/2022	NON-COMPETING CONTINUATION	$391,136
2022	2021	UNIVERSITY OF MIAMI	1400 NW 10TH AVE	MIAMI	FL	33136	MIAMI-DADE	USA	Vision Research	001	26	1/18/2022	COMPETING CONTINUATION	$98,360
2022	2020	UNIVERSITY OF MIAMI	1400 NW 10TH AVE	MIAMI	FL	33136	MIAMI-DADE	USA	Vision Research	000	25	1/18/2022	NON-COMPETING CONTINUATION	-$98,360
														Subtotal = $391,136

Issue Date FY: 2021 ( Subtotal = $403,232 )
2021	2021	UNIVERSITY OF MIAMI	1600 NW 10 AVE	MIAMI	FL	33136	MIAMI-DADE	USA	Vision Research	000	26	8/31/2021	COMPETING CONTINUATION	$403,232
														Subtotal = $403,232

Issue Date FY: 2020 ( Subtotal = $421,545 )
2020	2020	UNIVERSITY OF MIAMI	1600 NW 10 AVE	MIAMI	FL	33136	MIAMI-DADE	USA	Vision Research	000	25	8/25/2020	NON-COMPETING CONTINUATION	$421,545
														Subtotal = $421,545

Issue Date FY: 2019 ( Subtotal = $421,545 )
2019	2019	UNIVERSITY OF MIAMI	1600 NW 10 AVE	MIAMI	FL	33136	MIAMI-DADE	USA	Vision Research	000	24	8/30/2019	NON-COMPETING CONTINUATION	$421,545
														Subtotal = $421,545

Issue Date FY: 2018 ( Subtotal = $421,545 )
2018	2018	UNIVERSITY OF MIAMI	1600 NW 10 AVE	MIAMI	FL	33136	MIAMI-DADE	USA	Vision Research	000	23	8/20/2018	NON-COMPETING CONTINUATION	$421,545
														Subtotal = $421,545

Issue Date FY: 2017 ( Subtotal = $431,200 )
2017	2017	UNIVERSITY OF MIAMI	1600 NW 10 AVE	MIAMI	FL	33136	MIAMI-DADE	USA	Vision Research	002	22	8/28/2017	NON-COMPETING CONTINUATION	$431,200
2017	2016	UNIVERSITY OF MIAMI	1600 NW 10 AVE	MIAMI	FL	33136	MIAMI-DADE	USA	Vision Research	001	21	3/8/2017	COMPETING CONTINUATION	$118,309
2017	2015	UNIVERSITY OF MIAMI	1600 NW 10 AVE	MIAMI	FL	33136	MIAMI-DADE	USA	Vision Research	000	20	3/8/2017	NON-COMPETING CONTINUATION	-$118,309
														Subtotal = $431,200

Issue Date FY: 2016 ( Subtotal = $427,362 )
2016	2016	UNIVERSITY OF MIAMI	P.O. BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	000	21	8/9/2016	COMPETING CONTINUATION	$427,362
														Subtotal = $427,362

Issue Date FY: 2015 ( Subtotal = $376,075 )
2015	2015	UNIVERSITY OF MIAMI	P.O. BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	000	20	8/14/2015	NON-COMPETING CONTINUATION	$376,075
														Subtotal = $376,075

Issue Date FY: 2014 ( Subtotal = $376,075 )
2014	2014	UNIVERSITY OF MIAMI	P.O. BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	000	19	9/12/2014	NON-COMPETING CONTINUATION	$376,075
														Subtotal = $376,075

Issue Date FY: 2013 ( Subtotal = $383,750 )
2013	2013	UNIVERSITY OF MIAMI	P.O. BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	001	18	7/12/2013	COMPETING CONTINUATION	$383,750
2013	2012	UNIVERSITY OF MIAMI	POST OFFICE BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	000	17	7/9/2013	NON-COMPETING CONTINUATION	$0
														Subtotal = $383,750

Issue Date FY: 2012 ( Subtotal = $363,528 )
2012	2012	UNIVERSITY OF MIAMI	P.O. BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	000	17	4/20/2012	NON-COMPETING CONTINUATION	$363,528
														Subtotal = $363,528

Issue Date FY: 2011 ( Subtotal = $363,528 )
2011	2011	UNIVERSITY OF MIAMI	P.O. BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	000	16	5/13/2011	NON-COMPETING CONTINUATION	$363,528
														Subtotal = $363,528

Issue Date FY: 2010 ( Subtotal = $378,675 )
2010	2010	UNIVERSITY OF MIAMI	P.O. BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	000	15	4/19/2010	NON-COMPETING CONTINUATION	$378,675
														Subtotal = $378,675

Issue Date FY: 2009 ( Subtotal = $382,500 )
2009	2009	UNIVERSITY OF MIAMI	P.O. BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	001	14	4/23/2009	COMPETING CONTINUATION	$382,500
2009	2008	UNIVERSITY OF MIAMI	POST OFFICE BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	000	13	4/23/2009	NON-COMPETING CONTINUATION	$0
														Subtotal = $382,500

Issue Date FY: 2008 ( Subtotal = $419,045 )
2008	2008	UNIVERSITY OF MIAMI	POST OFFICE BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	000	13	3/3/2008	NON-COMPETING CONTINUATION	$419,045
														Subtotal = $419,045

Issue Date FY: 2007 ( Subtotal = $421,223 )
2007	2007	UNIVERSITY OF MIAMI	POST OFFICE BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	000	12	1/22/2007	NON-COMPETING CONTINUATION	$421,223
														Subtotal = $421,223

Issue Date FY: 2006 ( Subtotal = $419,967 )
2006	2006	UNIVERSITY OF MIAMI	POST OFFICE BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	001	11	2/24/2006	SUPPLEMENT FOR EXPANSION	$92,131
2006	2006	UNIVERSITY OF MIAMI	POST OFFICE BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	000	11	1/19/2006	NON-COMPETING CONTINUATION	$327,836
														Subtotal = $419,967

Issue Date FY: 2005 ( Subtotal = $335,725 )
2005	2005	UNIVERSITY OF MIAMI	POST OFFICE BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	000	10	11/10/2004	NON-COMPETING CONTINUATION	$335,725
														Subtotal = $335,725

Issue Date FY: 2004 ( Subtotal = $335,725 )
2004	2004	UNIVERSITY OF MIAMI	POST OFFICE BOX 248106	CORAL GABLES	FL	33124	MIAMI-DADE	USA	Vision Research	000	9	11/27/2003	COMPETING CONTINUATION	$335,725
														Subtotal = $335,725

Issue Date FY: 2002 ( Subtotal = $252,752 ) (Continued on the next page)
Grand Total All Awards = $9,483,832

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Setting the Stage for Replacement of Mitochondrial Genes

Award Number: R01EY010804

ORGANIZATION: NATIONAL EYE INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

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