Veenita Khare

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Anusha Sivakumar (1)Alexis N. Corl (1)Keneth Jordan Muñoz (1)Subhashini Arumugam (1)Betul Gunes (1)Angelyn B. Solis (1)Rishika Murthy (1)Lilas Rony El-Hachem (1)

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Oct 15, 2026

Anusha Sivakumar +183d ago

Edited HSPCs rescue frataxin deficiency and inflammation-driven multisystemic pathology in Friedreich's ataxia mice.

It is demonstrated that ex vivo CRISPR/Cas9 excision of the GAA expansion in hematopoietic stem and progenitor cells (HSPCs) provides durable, systemic therapy for Friedreich's ataxia and is established as a one-time, disease-modifying therapy for FRDA.

Anusha Sivakumar, Alexis N. Corl, Keneth Jordan Muñoz +16

Veenita Khare

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Veenita Khare

Publication activitypapers/week, last 8 weeks

Frequent co-authors

Papers (1)