Gene Therapy of Inherited Diseases Using CRISPR/Cas and Other Platforms
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Biology".
Deadline for manuscript submissions: 30 June 2024 | Viewed by 372
Special Issue Editor
Interests: nanoparticles; carcinogenic potential; lactoferrin; caner therapy
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
It is well known that CRISPR/Cas technology has the potential to advance the gene therapy field. Gene therapy includes ex vivo and in vivo genomic editing. There are numerous clinical trials in which CRISPR/Cas technology is used for ex vivo genomic editing. In these trials, the primary use of CRISPR/Cas is to directly disrupt gene expression or to disrupt the function of gene repressors to induce target gene expression. Similarly, the use of CRISPR/Cas for in vivo genomic editing in clinical trials is primarily to induce gene knockout. One trial (NCT05514249) attempted to use CRISPR/Cas in vivo genomic editing to restore the expression of dystrophin; however, the patient died. To date, productive use of CRISPR/Cas to directly repair gene expression in vivo has not been successfully established in clinical trials.
Currently, the most used platform for in vivo gene repair is adeno-associated viruses (AAVs). Although trials using AAV-mediated therapy to treat muscular dystrophy reported only temporary improvement, AAV-based therapy of gene deficiencies in the retina and infantile-onset spinal muscular atrophy type 1 appear to be successful. Zinc finger nucleases also appear to have been used successfully to treat patients with severe hemophilia B and mucopolysaccharidosis I and II.
In this Special Issue, we would like to emphasize the use of gene therapy in repairing genomic deficiencies. Suggested topics include, but are not limited to:
- The use of CRISPR/Cas to repair genomic deficiencies in vivo;
- Is it possible to use CRISPR/Cas to insert genetic material in vivo;
- The use of base and prime editors to repair genomic deficiencies in vivo;
- Fanzor, another RNA-guided endonuclease;
- The uses and limitations of adeno-associated virus-mediated repair genomic deficiencies in vivo;
- The uses and limitations of zinc finger nucleases to repair genomic deficiencies in vivo.
Dr. David B. Alexander
Guest Editor
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Keywords
- CRISPR/Cas technology
- gene therapy
- adeno-associated viruses (AAVs)
- spinal muscular atrophy
