Gene Therapy for Rare Diseases

Dear Colleagues,

Each rare disease is infrequent, but since more than 7000 rare diseases exist, more than 300 million patients are suffering from rare diseases worldwide, making them an important unmet health problem. Most rare diseases are caused by mutations. Therefore, gene therapy is the only curative treatment possibility for patients with rare diseases. Stimulated by the development of CRISPR gene editing and improved viral vector technologies, many trials are currently ongoing to correct the defective genes of patients with rare diseases and improve their quality of life. On the other hand, many challenges still need to be overcome to make gene therapy a standard treatment option in the clinic. These challenges include the efficient targeting of relevant stem cells, the effectiveness of precise genome editing, and patient safety. The recent development of prime editing and base editing has further contributed to the excitement in the field and to the hope that a cure for many rare diseases is possible in the near future. This Special Issue will accept reviews and original research articles in the field of gene editing and rare diseases.

Prof. Dr. Cord Brakebusch
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Cells is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2700 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

• gene therapy
• gene editing
• rare diseases
• CRISPR
• viral vectors
• base editing
• prime editing