Title:
SECRETED UBE3A FOR TREATMENT OF NEUROLOGICAL DISORDERS
Document Type and Number:
WIPO Patent Application WO/2022/272171
Kind Code:
A3
Abstract:
A novel vector, composition and method of treating a UBE3A deficiency disease is presented. A novel UBE3A vector construct was generated with an additional secretion sequence to allow the secretion from cells. This secreted only E6AP protein maintains its presence outside the cell and is capable of diffusing to greater distances to cover more of the brain and rescue disease pathology.
Inventors:
NASH KEVIN RON (US)
Application Number:
PCT/US2022/035166
Publication Date:
March 30, 2023
Filing Date:
June 27, 2022
Export Citation:
Assignee:
UNIV SOUTH FLORIDA (US)
International Classes:
A61K38/53; A61K48/00; A61P25/00; C12N9/00; C12N15/86
Domestic Patent References:
| WO2020191366A1 | 2020-09-24 |
Foreign References:
| US20200113955A1 | 2020-04-16 | |||
| US20190208752A1 | 2019-07-11 | |||
| US20180104358A1 | 2018-04-19 |
Attorney, Agent or Firm:
LAWSON, Michele L. (US)
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