Crispr-related methods and compositions
AssigneeEditas Medicine, Inc.
InventorsAlexandra GLUCKSMANN, Deborah PALESTRANT, Louis Anthony TARTAGLIA, Jordi MATA-FINK, Agnieszka Dorota CZECHOWICZ
Life of this patent
Estimating the term…
Abstract
Methods and compositions useful in targeting a payload to or editing a target nucleic acid utilizing CRISPR/Cas9 and guide RNA (gRNA) are disclosed herein
Claims24
A composition comprising a gRNA molecule comprising a targeting domain which is complementary with a target sequence from a target nucleic acid from a gene or pathway implicated in a disease or disorder listed in Table VII IX-2.
2-124. (canceled)
126-153. (canceled)
A method of altering a target nucleic acid of a cell implicated in a disease or disorder listed in Table IX-2, comprising contacting said cell with
- a composition comprising:
- 1a) one or more gRNA molecules;
- 1b) one or more Cas9 molecules;
- and
- 1c) optionally, a template nucleic acid;
- 2a) one or more gRNA molecules;
- 2b) one or more nucleic acids encoding one or more Cas9 molecules;
- and
- 2c) optionally, a template nucleic acid;
- 3a) one or more nucleic acids which encode one or more gRNA molecules;
- 3b) one or more Cas9 molecules;
- and
- 3c) optionally, a template nucleic acid;
- or
- 4a) one or more nucleic acids which encode one or more gRNA molecules;
- 4b) one or more nucleic acids encoding one or more Cas9 molecules;
- and
- 4c) optionally, a template nucleic acid.
155-180. (canceled)
A method of treating a subject by altering a target nucleic acid implicated in one or more of the diseases or disorders listed in Table IX-2, comprising administering to the subject, an effective amount of
- a composition comprising:
- 1a) one or more gRNA molecules;
- 1b) one or more Cas9 molecules;
- and
- 1c) optionally, a template nucleic acid;
- 2a) one or more gRNA molecules;
- 2b) one or more nucleic acids encoding one or more Cas9 molecules;
- and
- 2c) optionally, a template nucleic acid;
- 3a) one or more nucleic acids which encode one or more gRNA molecules;
- 3b) one or more Cas9 molecules;
- and
- 3c) optionally, a template nucleic acid;
- or
- 4a) one or more nucleic acids which encode one or more gRNA molecules;
- 4b) one or more nucleic acids encoding one or more Cas9 molecules;
- and
- 4c) optionally, a template nucleic acid.
182-258. (canceled)
Dependencies are inferred from the claim text.