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Related Concept Videos

CRISPR01:59

CRISPR

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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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Conservative Site-specific Recombination and Phase Variation02:53

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Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...
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Homologous Recombination02:31

Homologous Recombination

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The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...
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CRISPR and crRNAs02:53

CRISPR and crRNAs

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Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...
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Construction of Homozygous Mutants of Migratory Locust Using CRISPR/Cas9 Technology
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Construction of Homozygous Mutants of Migratory Locust Using CRISPR/Cas9 Technology

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CRISPR-mediated Ophthalmic Genome Surgery.

Galaxy Y Cho1,2, Yazeed Abdulla3, Jesse D Sengillo1,4

  • 1Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA.

Current Ophthalmology Reports
|October 3, 2017
PubMed
Summary

Clustered regularly interspaced short palindromic repeats (CRISPR) offers promising ophthalmic genome surgery for inherited retinal dystrophies. CRISPR-mediated therapies are advancing toward clinical trials, addressing unmet needs in treating eye conditions.

Keywords:
CRISPR-Casgenome surgeryinduced pluripotent stem cellsinherited retinal dystrophy

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Area of Science:

  • Ophthalmology
  • Genetics
  • Biotechnology

Background:

  • Clustered regularly interspaced short palindromic repeats (CRISPR) is a versatile genome engineering system.
  • CRISPR technology holds significant potential for clinical applications in treating various diseases.
  • Ophthalmic genome surgery using CRISPR is an emerging field with promising therapeutic avenues.

Purpose of the Study:

  • To review the development and application of CRISPR-mediated ophthalmic genome surgery.
  • To highlight recent advancements and potential clinical uses of CRISPR in ophthalmology.
  • To discuss the therapeutic prospects for inherited and acquired ophthalmic conditions.

Main Methods:

  • Review of current CRISPR techniques for ophthalmic applications.
  • Analysis of preclinical disease modeling and gene editing successes.
  • Examination of CRISPR's potential for treating inherited retinal dystrophies.

Main Results:

  • Diverse CRISPR techniques are under development for a range of ophthalmic conditions.
  • Preclinical studies and gene editing successes indicate CRISPR's potential efficacy for inherited eye diseases.
  • CRISPR-mediated genome surgery for Leber congenital amaurosis is nearing clinical trials.

Conclusions:

  • Inherited retinal dystrophies have limited treatment options currently.
  • CRISPR-mediated genome surgery presents a potential future solution for these unmet needs.
  • Advancements in CRISPR technology pave the way for novel ophthalmic therapies.