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Splice modulators target PMS1 to reduce somatic expansion of the Huntington's disease-associated CAG repeat.
Published in:
Nature Communications, 2024, v. 15, n. 1, p. 1, doi. 10.1038/s41467-024-47485-0
By:
- McLean, Zachariah L.;
- Gao, Dadi;
- Correia, Kevin;
- Roy, Jennie C. L.;
- Shibata, Shota;
- Farnum, Iris N.;
- Valdepenas-Mellor, Zoe;
- Kovalenko, Marina;
- Rapuru, Manasa;
- Morini, Elisabetta;
- Ruliera, Jayla;
- Gillis, Tammy;
- Lucente, Diane;
- Kleinstiver, Benjamin P.;
- Lee, Jong-Min;
- MacDonald, Marcy E.;
- Wheeler, Vanessa C.;
- Mouro Pinto, Ricardo;
- Gusella, James F.
Publication type:
Article
Somatic CAG expansion in Huntington's disease is dependent on the MLH3 endonuclease domain, which can be excluded via splice redirection.
Published in:
Nucleic Acids Research, 2021, v. 49, n. 7, p. 3907, doi. 10.1093/nar/gkab152
By:
- Roy, Jennie C L;
- Vitalo, Antonia;
- Andrew, Marissa A;
- Mota-Silva, Eduarda;
- Kovalenko, Marina;
- Burch, Zoe;
- Nhu, Anh M;
- Cohen, Paula E;
- Grabczyk, Ed;
- Wheeler, Vanessa C;
- Mouro Pinto, Ricardo
Publication type:
Article