CRISPR/Cas System: A Powerful Strategy to Improve Monogenic Human Diseases as Therapeutic Delivery; Current Applications and Challenges
The 5,000 to 8,000 monogenic diseases are inherited disorders leading to mutations in a single gene. These diseases usually appear in childhood and sometimes lead to morbidity or premature death. Although treatments for such diseases exist, gene therapy is considered an effective and targeted method and has been used in clinics for monogenic diseases since 1989. Monogenic diseases are good candidates for novel therapeutic technologies like gene editing approaches to repair gene mutations. Clustered regularly interspaced short palindromic repeats (CRISPR)-based systems, the pioneer and effective gene editing tool, are utilized for <i>ex vivo</i> and <i>in vivo</i> treatment of monogenic diseases. The current review provides an overview of recent therapeutic applications of CRISPR-based gene editing in monogenic diseases in <i>in vivo</i> and ex vivo models. Furthermore, this review consolidates strategies aimed at providing new treatment options with gene therapy, thereby serving as a valuable reference for advancing the treatment landscape for patients with monogenic disorders.
Authors
- Maryam Bazgiri
- Zeinab Yousefi-Najafabadi (ORCID: https://orcid.org/0000-0003-1512-5327)
- Fatemeh Saberi (ORCID: https://orcid.org/0000-0002-6225-5987)
- Sepideh Ahmadi (ORCID: https://orcid.org/0000-0002-6178-1168)
- Marzieh Sameni (ORCID: https://orcid.org/0000-0003-1699-3783)
- Mohammad Salehi (ORCID: https://orcid.org/0000-0002-3768-4325)
- Gholam Ali Kardar (ORCID: https://orcid.org/0000-0001-8475-0594)
- Zeinab Dehghan (ORCID: https://orcid.org/0000-0002-5976-9231)
- Vahid Jajarmi (ORCID: https://orcid.org/0000-0002-5080-7414)
- Rezvan Mohammadi (ORCID: https://orcid.org/0000-0002-0870-3080)
- Effat Noori (ORCID: https://orcid.org/0000-0002-8196-1967)
- Yong Teng (ORCID: https://orcid.org/0000-0002-1856-7289)
- Farzaneh Khani (ORCID: https://orcid.org/0000-0002-8711-6393)
- Parisa Moradbeigi
- Tayyebeh Pilehchi
- Forough Shams
- Zeinab Esmaeelzadeh
Institutions
- Shahed University (IR)
- Emory University (US)
- Shiraz University (IR)
- Shiraz University of Medical Sciences (IR)
- National Institute of Genetic Engineering and Biotechnology (IR)
- Shahid Beheshti University of Medical Sciences (IR)
- Tehran University of Medical Sciences (IR)
Publication Details
- Journal
- Current Gene Therapy
- Published
- 2025-01-09
- DOI
- https://doi.org/10.2174/0115665232345516241119070150
- Primary Topic
- CRISPR and Genetic Engineering
- Type
- article
- Field-Weighted Citation Impact
- 0.00