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AssigneesThe Broad Institute, Inc., Baylor College of Medicine, Vanderbilt University, President and Fellows of Harvard College
InventorsDavid R. Liu, Luke W. Koblan, Jonathan D. Brown, Charles Yang Lin
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The disclosure provides adenosine deaminases that are capable of deaminating adenosine in DNA to treat Hutchin-son-Gilford progeria syndrome (HOPS). The disclosure also provides fusion proteins, guide RNAs and compositions comprising a Cas9 (e.g., a Cas9 nickase) domain and adenosine deaminases that deaminate adenosine in DNA, for example in a LNA gene. In some embodiments, adenosine deaminases provided herein are used to correct a C1824T mutation in LMNA. In some embodiments, the methods and compositions provided herein are used to treat Hutchinson-Gilford progeria syndrome (HGPS).
A method for deaminating an adenosine nucleobase (A) in an LMNA gene, the method comprising contacting the LMNA gene with an adenosine base editor and a guide RNA bound to the adenosine base editor, wherein the guide RNA (gRNA) comprises a guide sequence that is complementary to a target nucleic acid sequence in the LMNA gene.
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A guide RNA comprising a guide sequence, wherein the guide sequence of the guide RNA comprises:
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A complex comprising (i) a base editor, and (ii) a guide RNA, wherein the guide RNA comprises a guide sequence comprising the nucleic acid sequence:
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Dependencies are inferred from the claim text.