A Review of CRISPR Tools for Treating Usher Syndrome: Applicability, Safety, Efficiency, and In Vivo Delivery.
A Review of CRISPR Tools for Treating Usher Syndrome: Applicability, Safety, Efficiency, and In Vivo Delivery.
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DOI：
10.3390/ijms24087603
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发表时间：
2023-04-20
影响因子：
5.6
通讯作者：
MacLaren, Robert E.
中科院分区：
文献类型：
--
作者：
Major, Lauren;McClements, Michelle E.;MacLaren, Robert E.
这篇综述考虑了对Usher综合征治疗的研究，Usher综合征是一种以常染色体隐性遗传方式遗传的眼盲综合征。Usher综合征突变是显著异质性的，涉及许多不同的基因，并且由于患者人群很少，研究赠款有限。此外，基因扩增疗法是不可能的，但三个Usher综合征的cDNA序列超过4.7 kb的AAV包装限制。因此，至关重要的是将研究工作重点放在具有最广泛适用性的替代工具上。CRISPR领域在2012年发现Cas9的DNA编辑活性后，近年来迅速发展。新一代的CRISPR工具继承了最初的CRISPR/Cas9模型，能够进行更复杂的基因组修改，如表观遗传修饰和精确的序列改变。这篇综述将评估迄今为止最流行的CRISPR工具：CRISPR/Cas9、碱基编辑和引物编辑。它将考虑这些工具的适用性（与十种最常见的USH 2A突变有关），安全性，效率和体内递送潜力，旨在指导未来的研究投资。
This review considers research into the treatment of Usher syndrome, a deaf-blindness syndrome inherited in an autosomal recessive manner. Usher syndrome mutations are markedly heterogeneous, involving many different genes, and research grants are limited due to minimal patient populations. Furthermore, gene augmentation therapies are impossible in all but three Usher syndromes as the cDNA sequence exceeds the 4.7 kb AAV packaging limit. It is, therefore, vital to focus research efforts on alternative tools with the broadest applicability. The CRISPR field took off in recent years following the discovery of the DNA editing activity of Cas9 in 2012. New generations of CRISPR tools have succeeded the original CRISPR/Cas9 model to enable more sophisticated genomic amendments such as epigenetic modification and precise sequence alterations. This review will evaluate the most popular CRISPR tools to date: CRISPR/Cas9, base editing, and prime editing. It will consider these tools in terms of applicability (in relation to the ten most prevalent USH2A mutations), safety, efficiency, and in vivo delivery potential with the intention of guiding future research investment.
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影响因子：
16.6
作者：
Chen L;Park JE;Paa P;Rajakumar PD;Prekop HT;Chew YT;Manivannan SN;Chew WL
通讯作者：
Chew WL
影响因子：
5.9
作者：
Assawachananont, Juthaporn;Mandai, Michiko;Okamoto, Satoshi;Yamada, Chikako;Eiraku, Mototsugu;Yonemura, Shigenobu;Sasai, Yoshiki;Takahashi, Masayo
通讯作者：
Takahashi, Masayo
影响因子：
16.6
作者：
通讯作者：
--
影响因子：
2.9
作者：
Battacharya, G;Cosgrove, D
通讯作者：
Cosgrove, D
影响因子：
4.6
作者：
Hashimoto M;Takemoto T
通讯作者：
Takemoto T