Multiplexed Genome Editing for Efficient Phenotypic Screening in Zebrafish

Guo, Shuyu; Gao, Ge; Zhang, Cuizhen; Peng, Gang

doi:10.3390/vetsci9020092

Cited by 3 publications

(4 citation statements)

References 43 publications

(49 reference statements)

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“…Zebrafish is also widely used to investigate candidate genes for human genetic HL. Recently, based on CRISPR/Cas9 system, Gou et al proposed a novel multiplex genome editing strategy that could simultaneously target five genes and rapidly generate individual homozygous zebrafish mutants for functional genetics research [ 163 ]. According to the results of the C-start assay and the AMI-43 staining, a new gene mutation ( tmem183a ) was identified to be associated with HL, which may affect the normal state of mechanoelectrical transduction channels in hair cells [ 163 ].…”

Section: Crispr/cas9 In Genetic Hearing Lossmentioning

confidence: 99%

“…Recently, based on CRISPR/Cas9 system, Gou et al proposed a novel multiplex genome editing strategy that could simultaneously target five genes and rapidly generate individual homozygous zebrafish mutants for functional genetics research [ 163 ]. According to the results of the C-start assay and the AMI-43 staining, a new gene mutation ( tmem183a ) was identified to be associated with HL, which may affect the normal state of mechanoelectrical transduction channels in hair cells [ 163 ]. By linkage analysis and exome sequencing, Rodrigo et al identified a rare missense variant (c.2810C > G) in the NCOA3 gene as the best candidate to be causative of bilateral, progressive, non-syndromic, and sensorineural HL in a large Brazilian family with autosomal dominant inheritance [ 164 ].…”

Section: Crispr/cas9 In Genetic Hearing Lossmentioning

confidence: 99%

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The applications of CRISPR/Cas-mediated genome editing in genetic hearing loss

Tao

Deng

et al. 2023

Cell Biosci

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Hearing loss (HL) can be caused by a number of different genetic factors. Non-syndromic HL refers that HL occurs as an isolated symptom in an individual, whereas syndromic HL refers that HL is associated with other symptoms or abnormalities. To date, more than 140 genes have been identified as being associated with non-syndromic HL, and approximately 400 genetic syndromes can include HL as one of the clinical symptoms. However, no gene therapeutic approaches are currently available to restore or improve hearing. Therefore, there is an urgent necessity to elucidate the possible pathogenesis of specific mutations in HL-associated genes and to investigate the promising therapeutic strategies for genetic HL. The development of the CRISPR/Cas system has revolutionized the field of genome engineering, which has become an efficacious and cost-effective tool to foster genetic HL research. Moreover, several in vivo studies have demonstrated the therapeutic efficacy of the CRISPR/Cas-mediated treatments for specific genetic HL. In this review, we briefly introduce the progress in CRISPR/Cas technique as well as the understanding of genetic HL, and then we detail the recent achievements of CRISPR/Cas technique in disease modeling and therapeutic strategies for genetic HL. Furthermore, we discuss the challenges for the application of CRISPR/Cas technique in future clinical treatments.

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Section: Crispr/cas9 In Genetic Hearing Lossmentioning

confidence: 99%

Section: Crispr/cas9 In Genetic Hearing Lossmentioning

confidence: 99%

The applications of CRISPR/Cas-mediated genome editing in genetic hearing loss

Tao

Deng

et al. 2023

Cell Biosci

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“…The gRNA targeted sequence (5 0 -GATCCTGAACGGACA-CATCGTGG-3 0 ) was located in the sixth exon of the ap4s1 gene. The gRNA template was constructed from oligonucleotides listed in Table 1 using a de novo cloning-free method (Guo et al, 2022). Microinjections were performed in one-cell stage embryos of WT zebrafish.…”

Section: Generation Of Ap4s1 Mutantmentioning

confidence: 99%

Ap4s1 truncation leads to axonal defects in a zebrafish model of spastic paraplegia 52

Li,

Zhang,

Peng

2023

Intl J of Devlp Neuroscience

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Biallelic mutations in AP4S1, the σ4 subunit of the adaptor protein complex 4 (AP‐4), lead to autosomal recessive spastic paraplegia 52 (SPG52). It is a subtype of AP‐4‐associated hereditary spastic paraplegia (AP‐4‐HSP), a complex childhood‐onset neurogenetic disease characterized by progressive spastic paraplegia of the lower limbs. This disease has so far lacked effective treatment, in part due to a lack of suitable animal models. Here, we used CRISPR/Cas9 technology to generate a truncation mutation in the ap4s1 gene in zebrafish. The ap4s1 truncation led to motor impairment, delayed neurodevelopment, and distal axonal degeneration. This animal model is useful for further research into AP‐4 and AP‐4‐HSP.

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“…Over 70% of human genes have at least one zebrafish orthologue [ 36 ], with the Xenopus genome including orthologs of ~80% of human disease genes [ 37 ]. More recently, methods to increase the genome editing efficiency of the Clustered Regularly Interspaced Short Palindromic Repeat– (CRISPR–) Cas9 system in zebrafish [ 38 , 39 , 40 , 41 , 42 , 43 ] and Xenopus [ 44 , 45 , 46 , 47 , 48 ] have led to new human disease models.…”

Section: Aquatic Freshwater Vertebrate Animal Model Advantagesmentioning

confidence: 99%

Aquatic Freshwater Vertebrate Models of Epilepsy Pathology: Past Discoveries and Future Directions for Therapeutic Discovery

Williams¹,

Mruk²

2022

IJMS

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Epilepsy is an international public health concern that greatly affects patients’ health and lifestyle. About 30% of patients do not respond to available therapies, making new research models important for further drug discovery. Aquatic vertebrates present a promising avenue for improved seizure drug screening and discovery. Zebrafish (Danio rerio) and African clawed frogs (Xenopus laevis and tropicalis) are increasing in popularity for seizure research due to their cost-effective housing and rearing, similar genome to humans, ease of genetic manipulation, and simplicity of drug dosing. These organisms have demonstrated utility in a variety of seizure-induction models including chemical and genetic methods. Past studies with these methods have produced promising data and generated questions for further applications of these models to promote discovery of drug-resistant seizure pathology and lead to effective treatments for these patients.

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Multiplexed Genome Editing for Efficient Phenotypic Screening in Zebrafish

Cited by 3 publications

References 43 publications

The applications of CRISPR/Cas-mediated genome editing in genetic hearing loss

The applications of CRISPR/Cas-mediated genome editing in genetic hearing loss

Ap4s1 truncation leads to axonal defects in a zebrafish model of spastic paraplegia 52

Aquatic Freshwater Vertebrate Models of Epilepsy Pathology: Past Discoveries and Future Directions for Therapeutic Discovery

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