RAiD 10.26259/ba5702c4 A novel antisense oligonucleotide therapeutic to restore defective splicing in ALS
Dates
Start Date
31-Jul-2024
End Date
31-Dec-2024
Titles
Title
A novel antisense oligonucleotide therapeutic to restore defective splicing in ALS
Title Type
Primary
Title Type: Primary Preferred full description or abstract.
Start Date
31-Jul-2024
End Date
---
Language
English
Descriptions
Description
The purpose of this project is to take a precision medicine approach and design a drug that is specifically targeted to correct mutations that are present in the ALS gene KIF5A. Drug sequences will be screened in stem cells from an ALS patient harbouring a KIF5A mutation that results in the skipping of exon 27 (part of the protein coding sequence). The goal of this project is to restore the expression of full length KIF5A protein in patient cells.
Description Type
Primary
Description Type: Primary Preferred full description or abstract
Language
English
Contributors
Contributor
https://orcid.org/0000-0002-7092-8344
Leader
Yes
Contact
Yes
Positions
Position
Principal or Chief Investigator
Position: Principal or Chief Investigator Principal investigator refers to the person(s) in charge of a research project
Start Date
31-Jul-2024
End Date
---
Roles
Conceptualization
Organisations
Organisation ID
https://ror.org/02stey378
Roles
Role
Lead Research Organisation
Role: Lead Research Organisation The research organistion administratively responsible for the project; administering organisation
Start Date
2024-07-31
End Date
---
RelatedObjects
No Entries
Alternate Identifier
No Entries
Alternate URLs
No Entries
Related RAiDs
No Entries
Access
Type
Open Access
Type: Open Access Open access refers to a resource that is immediately and permanently online, and free for all on the Web, without financial and technical barriers.The resource is either stored in the repository or referenced to an external journal or trustworthy archive.
Language
eng
Text
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Embargo Expiry
2025-08-08
Subjects
Subject
Neurology and neuromuscular diseases (320905)
Keywords
Text
Subject keyword example text...
Language
eng
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