In Vivo CRISPR/Cas9-Mediated Genome Editing Mitigates Photoreceptor Degeneration in a Mouse Model of X-Linked Retinitis Pigmentosa

Hu, Shuang; Du, Juan; Chen, Ningning; Jia, Ruixuan; Zhang, Jinlu; Liu, Xiaozhen; Yang, Liping

doi:10.1167/iovs.61.4.31

Cited by 31 publications

(21 citation statements)

References 40 publications

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“…Since the first report of AAV-based CRISPR-Cas9 delivery for in vivo genome editing, it has made exciting progress in numerous disease models, including blood disorders 50 , 51 , 52 , metabolic liver diseases 44 , 53 , 54 , 55 , muscular diseases 56 , 57 , 58 , and ocular diseases 59 , 60 . In 2020, in a landmark clinical trial sponsored by Editas Medicines (NCT03872479), AAV became the very first CRISPR-Cas9 delivery vector for direct injection into a patient of Leber congenital amaurosis type 10.…”

Section: Current Vectors For In Vivo Delivery Of Crispr-cas9 Therapeuticsmentioning

confidence: 99%

In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges

Behr

Zhou²,

et al. 2021

Acta Pharmaceutica Sinica B

123

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Within less than a decade since its inception, CRISPR-Cas9-based genome editing has been rapidly advanced to human clinical trials in multiple disease areas. Although it is highly anticipated that this revolutionary technology will bring novel therapeutic modalities to many diseases by precisely manipulating cellular DNA sequences, the low efficiency of in vivo delivery must be enhanced before its therapeutic potential can be fully realized. Here we discuss the most recent progress of in vivo delivery of CRISPR-Cas9 systems, highlight innovative viral and non-viral delivery technologies, emphasize outstanding delivery challenges, and provide the most updated perspectives.

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Section: Current Vectors For In Vivo Delivery Of Crispr-cas9 Therapeuticsmentioning

confidence: 99%

In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges

Behr

Zhou²,

et al. 2021

Acta Pharmaceutica Sinica B

123

View full text Add to dashboard Cite

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“…AAV-mediated gene transfer to treat retinitis pigmentosa has already been approved as the first AAV gene therapy in history, 42 but basic studies on CRISPR-based genome editing for this purpose only started in 2016. Since then, many CRISPR-based approaches, each targeting a different gene ( Nrl 43 44 Mertk , 45 Pde6b , 46 47 Rho , 48 49 and RPGR 50 ), have achieved success in animal models. Specifically, the Nrl gene was depleted via NHEJ or repressed via an approach called CRISPR interference, both instigated by AAV vector delivery of CRISPR components, and the Mertk gene was corrected with a novel method called homology-independent targeted integration.…”

Section: Clinical Applications Of Crispr-based Genome Editingmentioning

confidence: 99%

Basic Principles and Clinical Applications of CRISPR-Based Genome Editing

Lim

Kim

2022

Yonsei Med J

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Advances in sequencing technologies have facilitated the discovery of previously unknown genetic variants in both inherited and acquired disorders, and tools to correct these pathogenic variants are rapidly evolving. Since the first introduction of CRISPR-Cas9 in 2012, the field of CRISPR-based genome editing has progressed immensely, giving hope to many patients suffering from genetic disorders that lack effective treatment. In this review, we will examine the basic principles of CRISPR-based genome editing, explain the mechanisms of new genome editors, including base editors and prime editors, and evaluate the therapeutic possibilities of CRISPR-based genome editing by focusing on recently published clinical trials and animal studies. Although efficacy and safety issues remain a large concern, we cannot deny that CRISPR-based genome editing will soon be prevalent in clinical practice.

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“…for retinal gene editing and achieved high editing effects in the adult mouse retina [65]. Studies have also demonstrated successful AAV-based CRISPR/Cas9 gene editing in the retina of retinal degeneration mouse model [66,67]. AAV-based delivery of CRISPR/Cas components has also been used to knockdown IGF in the central nervous system [68].…”

Section: Adeno-associated Virusmentioning

confidence: 99%

Approach for in vivo delivery of CRISPR/Cas system: a recent update and future prospect

Chuang

Phipps

Lin

et al. 2021

Cell. Mol. Life Sci.

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The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system provides a groundbreaking genetic technology that allows scientists to modify genes by targeting specific genomic sites. Due to the relative simplicity and versatility of the CRISPR/Cas system, it has been extensively applied in human genetic research as well as in agricultural applications, such as improving crops. Since the gene editing activity of the CRISPR/Cas system largely depends on the efficiency of introducing the system into cells or tissues, an efficient and specific delivery system is critical for applying CRISPR/Cas technology. However, there are still some hurdles remaining for the translatability of CRISPR/Cas system. In this review, we summarized the approaches used for the delivery of the CRISPR/Cas system in mammals, plants and aquaculture. We further discussed the aspects of delivery that can be improved to elevate the potential for CRISPR/Cas translatability.

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In Vivo CRISPR/Cas9-Mediated Genome Editing Mitigates Photoreceptor Degeneration in a Mouse Model of X-Linked Retinitis Pigmentosa

Abstract: In vivo CRISPR/Cas9-mediated genome editing mitigates photoreceptor degeneration in a mouse model of X-linked retinitis pigmentosa. Invest

Cited by 31 publications

References 40 publications

In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges

In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges

Basic Principles and Clinical Applications of CRISPR-Based Genome Editing

Approach for in vivo delivery of CRISPR/Cas system: a recent update and future prospect

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