Correcting monogenic skin diseases in situ: Assessing the potential of topically applicable gene therapy.

原位纠正单基因皮肤病：评估局部适用基因疗法的潜力。

• 批准号: 400904
• 负责人: Hedtrich Sarah
• 金额: $ 34.44万
• 依托单位: University of British Columbia
• 依托单位国家: 加拿大
• 项目类别: Operating Grants
• 财政年份: 2019
• 资助国家: 加拿大
• 起止时间: 2019-03-01 至 2022-03-01
• 项目状态: 已结题
• 来源: https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=400904&lang=en
• 关键词: 3d Skin Disease Models; Autosomal Recessive Congenital Ichthyosis; Crispr-Cas9; Gene Editing; Monogenic Skin Disease; Nanocarriers For Topical Drug Delivery; Novel Treatment Options For Orphan Disease; Topical Delivery Of Biomacromolecules

Monogenic skin diseases such as autosomal recessive congenital ichthyosis (ARCI) are caused by mutations in a single gene. If these mutations could be corrected, the disease should be mitigated. Indeed, for recessively inherited conditions such as ARCI, c

单基因皮肤病，如常染色体隐性先天性鱼鳞病（ARCI）是由单个基因突变引起的。如果这些突变能够得到纠正，这种疾病就应该得到缓解。事实上，对于隐性遗传条件，如ARCI， c