Advances in Genome Editing for Genetic Hearing Loss
| Autor: | Xue Gao, Ning Ding, Matan Lieber-Kotz, Jie Yang, Sangsin Lee |
|---|---|
| Jazyk: | angličtina |
| Rok vydání: | 2020 |
| Předmět: |
Hearing loss
Genetic enhancement Pharmaceutical Science 02 engineering and technology Computational biology Biology Gene delivery Congenital hearing loss Polymorphism Single Nucleotide Article 03 medical and health sciences Sensory impairment Genome editing medicine otorhinolaryngologic diseases CRISPR Clustered Regularly Interspaced Short Palindromic Repeats Hearing Loss Gene 030304 developmental biology Gene Editing 0303 health sciences Genetic Therapy 021001 nanoscience & nanotechnology medicine.symptom 0210 nano-technology |
| Zdroj: | Adv Drug Deliv Rev |
| Popis: | According to the World Health Organization, hearing loss affects over 466 million people worldwide and is the most common human sensory impairment. It is estimated that genetic factors contribute to the causation of approximately 50% of congenital hearing loss. Yet, curative approaches to reversing or preventing genetic hearing impairment are still limited. The clustered regularly interspaced short palindromic repeats-associated protein 9 (CRISPR-Cas9) systems enable programmable and targeted gene editing in highly versatile manners and offer new gene therapy strategies for genetic hearing loss. Here, we summarize the most common deafness-associated genes, illustrate recent strategies undertaken by using CRISPR-Cas 9 systems for targeted gene editing and further compare the CRISPR strategies to non-CRISPR gene therapies. We also examine the merits of different vehicles and delivery forms of genome editing agents. Lastly, we describe the development of animal models that could facilitate the eventual clinical applications of the CRISPR technology to the treatment of genetic hearing diseases. |
| Databáze: | OpenAIRE |
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