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Tissue-Specific Gene Editing via Programmable Sendai -based Vectors (PSVs)

Award Number: F30HL184849
ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: FELLOWSHIP/SCHOLARSHIP/STUDENT LOANS
PERIOD OF PERFORMANCE START DATE: 03/12/2027
PERIOD OF PERFORMANCE END DATE: 03/11/2028

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Tissue-Specific Gene Editing via Programmable Sendai -based Vectors (PSVs) - Project Summary Recent breakthroughs in gene editing have enabled precise genetic correction for inherited and acquired diseases; however, therapeutic success relies heavily on safe, efficient delivery of editing tools to targeted cells. Viral vectors, particularly adeno-associated viruses (AAVs), have shown clinical promise but are limited by pre- existing immunity, small payload capacity, and restricted tissue targeting. To address these limitations, we propose developing a Programmable Sendai virus-based Vector (PSV) that integrates advanced prime editing technology with microRNA (miRNA)-guided restriction. Unlike traditional editing approaches, prime editors introduce precise genomic changes without double-strand DNA breaks, minimizing cytotoxicity. Leveraging the unique biology of PSV, which is neither pathogenic nor capable of genomic integration, we aim to develop a safer alternative to current gene delivery methods. PSV also supports delivery of large genetic payloads, making it well-suited for therapeutic applications. Here, we propose to use this platform to selectively edit the DNA of hematopoietic stem cells (HSCs) derived from peripheral blood mononuclear cells (PBMCs) ex vivo. As a clinically relevant proof-of-concept, this project focuses on Paroxysmal Nocturnal Hemoglobinuria (PNH), a severe blood disorder resulting from somatic mutations in the PIGA gene. Current treatments do not correct the underlying genetic defect and significantly reduce patient quality of life, highlighting a clear unmet clinical need. Our research aims to (1) Engineer and validate a PSV expressing prime editor (PSV-PE) targeting the PIGA locus in hematopoietic and pluripotent cell models. (2) Incorporate miRNA target sites into the PSV backbone to achieve stringent restriction of vector expression exclusively to HSCs. (3) Demonstrate targeted gene editing using PSV-PE vectors in mixed populations of primary PBMCs, including PNH patient-derived samples. This approach will validate a novel, safe, and scalable gene-editing platform broadly applicable to hematologic disorders driven by single-gene mutations, substantially advancing clinical gene therapy methods moving forward.


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: 2026 ( Subtotal = $55,114 )
2026	2026	NEW YORK UNIVERSITY	550 1ST AVE	NEW YORK	NY	10016	NEW YORK	USA	Blood Diseases and Resources Research	000	1	9/17/2026	NEW	$55,114
														Subtotal = $55,114

Grand Total All Awards = $55,114

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Tissue-Specific Gene Editing via Programmable Sendai -based Vectors (PSVs)

Award Number: F30HL184849

ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: FELLOWSHIP/SCHOLARSHIP/STUDENT LOANS

PERIOD OF PERFORMANCE START DATE: 03/12/2027

PERIOD OF PERFORMANCE END DATE: 03/11/2028

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