..., the cost-effective, rapid and higher throughput nature of Xenopus make it an essential member of the model organism armamentarium for understanding gene function in development and in relation to disease. As discussed previously, CRISPR/Cas9-based editing has become a popular approach...

.... Here, we used cerebellar organoids differentiated from human induced pluripotent stem cells combined with CRISPR/Cas9 gene editing to investigate the earliest molecular and cellular consequences of PTCH1 mutations on human cerebellar development. Our findings demonstrate that developmental mechanisms...

... and disease phenotypes. When combined with live imaging, they enable direct and repeated observation of cell behaviors within a developing retinal tissue. Here, we generated a human cone photoreceptor reporter line by CRISPR/Cas9 genome editing of WTC11-mTagRFPT-LMNB1 human induced pluripotent stem cells...

... to study neutrophils and macrophages. We used CRISPR-Cas9 and CRISPR-Cpf1, with oligonucleotide-directed homologous repair, to engineer zebrafish equivalents of the two most prevalent human APDS1 disease mutations. These zebrafish pik3cd alleles dominantly caused excessive neutrophilic inflammation...

... of the phenotype became complex with transitory phenotypes that are corrected over time. We also demonstrated that multiple-guide-derived CRISPR/Cas9 F0 ‘crispant’ embryos recapitulate zygotic null phenotypes, and rapidly identified ciliary phenotypes in 11 cilia-associated gene candidates ( ankfn1 , ccdc65...

... consequences of PLCB4 variants on EDNRA signaling is not known. Here, we show, using multiple signaling reporter assays, that known PLCB4 variants resulting from missense mutations exert a dominant-negative interference over EDNRA signaling. In addition, using CRISPR/Cas9, we find that F 0 mouse embryos...

... engineer disease-associated inversions, deletions and translocations in model systems. The emergence and refinement of genome editing technologies, such as CRISPR, have significantly expanded our ability to generate and interrogate chromosomal aberrations to better understand the networks that govern...

...-associated IL2RG gene. CRISPR Syrian hamster Adenovirus Animal model Knockout The rising popularity of the hamster model prompted the generation of much needed reagents and the development of genetically engineered hamster strains. The techniques to produce hamsters with specific gene...

... lipid accumulation and metabolic homeostasis was unaffected in mice fed a high-fat diet for 4 weeks. We also investigated the consequences of BSCL2 ablation in the human hepatocyte HepG2 cell line using CRISPR/Cas9 genome editing. No significant increases in lipid accumulation were observed in BSCL2...

... kidney development and congenital kidney diseases. Xenopus Nephron CAKUT Nephronophthisis PKD CRISPR Kidney NIDDK 10.13039/100000062 K01DK092320 R03 DK118771 University of Texas Health Science Center at Houston 10.13039/100012615 References Abdelhak...

... for creating insertion alleles ( Yoshimi et al., 2016 ). More recent reports show that co-injecting lssDNA donors with commercially available CRISPR ribonucleoprotein complexes (instead of the previous formats of Cas9 mRNA and sgRNAs), offers a highly robust and efficient strategy for insertion alleles...

... fraction and fraction shortening. In conclusion, this novel rabbit DMD model generated with the CRISPR/Cas9 system mimics the histopathological and functional defects in DMD patients, and could be valuable for preclinical studies. This article has an associated First Person interview with the first author...

...Sergey V. Prykhozhij; Shelby L. Steele; Babak Razaghi; Jason N. Berman ABSTRACT Clustered regularly interspaced palindromic repeats (CRISPR)/Cas-based adaptive immunity against pathogens in bacteria has been adapted for genome editing and applied in zebrafish ( Danio rerio ) to generate frameshift...

...’, providing a summary of the articles published in this launch issue and a flavour of what's to come, as well as highlighting the impact rat models have made and continue to make on understanding human disease. CRISPR Genomics Model organism Rat Translational The rat is one of the premier...