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
Access statement example text...
Embargo Expiry
2025-08-08

Subjects

Subject
Neurology and neuromuscular diseases (320905)
Keywords
Text
Subject keyword example text...
Language
eng

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