The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Aug. 02, 2022
Filed:
Sep. 23, 2021
Applicant:
University of Massachusetts, Boston, MA (US);
Inventor:
Miguel Sena Esteves, Westford, MA (US);
Assignee:
University of Massachusetts, Boston, MA (US);
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/86 (2006.01); A61P 35/00 (2006.01); C07K 14/47 (2006.01); A61K 48/00 (2006.01);
U.S. Cl.
CPC ...
C12N 15/86 (2013.01); A61P 35/00 (2018.01); C07K 14/4705 (2013.01); A61K 48/00 (2013.01); C12N 2750/14143 (2013.01);
Abstract
Aspects of the disclosure relate to compositions and methods of treating certain genetic disease (e.g., Neurofibromatosis type I) by delivering functional neurofibromin 1 (NF1) protein (e.g., mini-NF1 protein and/or full-length NF1 protein) to target cell (e.g., cells and/or tissue of a subject). The disclosure is based, in part, on isolated nucleic acids (e.g., rAAV vectors) and rAAVs engineered to express a functional NF1 protein (e.g., mini-NF1 protein and/or full-length NF1 protein) or variants thereof.