Patent · US Active

Compositions and methods for treatment of disorders related to CEP290

US10301366B2 · kind B2 · utility

1Cited by

0References

19Claims

0Family size

Assignee

The Trustees of the University of Pennsylvania · US

Inventors

Theodore G. Drivas · Philadelphia, US
Jean Bennett · Via Enrico Fermi, US

Key dates

Filing date	Nov 5, 2018
Grant date	May 28, 2019
Priority date	—
Expiry date	Nov 5, 2038

Classification

Technology area (CPC C)Chemistry; Metallurgy
CPC primaryC12Q2600/156
WIPO fieldPharmaceuticals
WIPO sectorChemistry

Abstract

Compositions are provided that comprise a recombinant vector carrying a nucleic acid sequence encoding a fragment of CEP290 lacking all or part of its N-terminal and C-terminal inhibitory regions, under the control of regulatory sequences which express the product of said gene in a selected cell of a mammalian subject, and a pharmaceutically acceptable carrier. These and other compositions are disclosed with are useful in methods for treating a mammalian subject having a disease associated with a CEP290 mutation, such as Lebers Congenital Amaurosis.

Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.