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CRISPR-mediated genome editing and human diseases

Cai, Liquan, Fisher, Alfred L., Huang, Haochu, Xie, Zijian·2016·Genes & Diseases
DOI 10.1016/j.gendis.2016.07.003PMC PMC6150104
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Fig. 1 CRISPR working mechanism. Guide RNA hybridizes with 20bp genomic DNA sequence and directs Cas9 endonuclease (colored in pink) to generate a double strand break which is usually located between 16 and 17bp region in the target sequence. Subsequently, DNA Mutagenesis is generated from DNA repair process, through either the non-homologous end-joining (NHEJ) or the homology-directed repair (HDR) mechanism. The final mutation could include insertion or deletion with several base pairs of DNA sequences (NHEJ pathway), or replacement with a particular DNA sequence used as a marker for further study (encoding for a fluorescence protein, tag protein, antibiotics, or the recognition sequence for a restriction enzyme digestion).
Fig. 1 CRISPR working mechanism. Guide RNA hybridizes with 20bp genomic DNA sequence and directs Cas9 endonuclease (colored in pink) to generate a double strand break which is usually located between 16 and 17bp region in the target sequence. Subsequently, DNA Mutagenesis is generated from DNA repair process, through either the non-homologous end-joining (NHEJ) or the homology-directed repair (HDR) mechanism. The final mutation could include insertion or deletion with several base pairs of DNA sequences (NHEJ pathway), or replacement with a particular DNA sequence used as a marker for further study (encoding for a fluorescence protein, tag protein, antibiotics, or the recognition sequence for a restriction enzyme digestion).CC BY-NC-ND · PubMed Central
Fig. 2 CRISPR engineering for the clinics. Advances in DNA sequencing technology make it easier to identify the disease-driving genetic mutations. Meanwhile, the patient-derived iPS cells have been established to model human diseases and drug discovery in vitro. The deteriorating mutations can be corrected via the use of CRISPR-mediated gene editing, and the modified cells can be then utilized as patient-specific medicine.
Fig. 2 CRISPR engineering for the clinics. Advances in DNA sequencing technology make it easier to identify the disease-driving genetic mutations. Meanwhile, the patient-derived iPS cells have been established to model human diseases and drug discovery in vitro. The deteriorating mutations can be corrected via the use of CRISPR-mediated gene editing, and the modified cells can be then utilized as patient-specific medicine.CC BY-NC-ND · PubMed Central

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Metadata

Authors
Cai, L., Fisher, A. L., Huang, H., & Xie, Z.
ORCID
Venue
Genes & Diseases
Publisher
Elsevier BV
Published
2016-12
Volume
3
Issue
4
Pages
244-251
ISSN
2352-3042
DOI
10.1016/j.gendis.2016.07.003
PMC
PMC6150104
Cited by
83
References
15
Language
en
Licence
Funders
  • National Institutes of Health
  • National Institute of Allergy and Infectious Diseases (AG013319, AG044271, AG044768, ES017761)

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