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Related Concept Videos

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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CRISPR01:59

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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 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.
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Genomics02:02

Genomics

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Genomics is the science of genomes: it is the study of all the genetic material of an organism. In humans, the genome consists of information carried in 23 pairs of chromosomes in the nucleus, as well as mitochondrial DNA. In genomics, both coding and non-coding DNA is sequenced and analyzed. Genomics allows a better understanding of all living things, their evolution, and their diversity. It has a myriad of uses: for example, to build phylogenetic trees, to improve productivity and...
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Genomic Imprinting and Inheritance02:30

Genomic Imprinting and Inheritance

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Diploid organisms inherit genetic material through chromosomes from both parents. Copies of the same gene are known as alleles. In most cases, both alleles are simultaneously expressed and allow various cellular processes to function optimally. If one of the alleles is missing or mutated, the expression of the other allele can compensate; however, this is not true for all genes.
The expression of some genes depends on which parent passed the gene to the offspring, through a phenomenon known as...
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Genome Size and the Evolution of New Genes03:21

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While every living organism has a genome of some kind (be it RNA, or DNA), there is considerable variation in the sizes of these blueprints. One major factor that impacts genome size is whether the organism is prokaryotic or eukaryotic. In prokaryotes, the genome contains little to no non-coding sequence, such that genes are tightly clustered in groups or operons sequentially along the chromosome. Conversely, the genes in eukaryotes are punctuated by long stretches of non-coding sequence.
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Updated: Feb 5, 2026

Genome Engineering of Primary Human B Cells Using CRISPR/Cas9
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CRISPR/Cas9 genome surgery for retinal diseases.

Christine L Xu1, Karen Sophia Park1, Stephen H Tsang2

  • 1Edward S Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA; Jonas Children's Vision Care and the Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University, New York, NY, USA.

Drug Discovery Today. Technologies
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Summary

Clustered regularly interspaced short palindromic repeats (CRISPR) gene editing offers new ways to model and treat retinal diseases. CRISPR/Cas9 technology may provide a one-time genetic surgery to correct mutations, improving patient outcomes.

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Area of Science:

  • Ophthalmology
  • Genetics
  • Molecular Biology

Background:

  • Retinal diseases cause significant patient burden.
  • Gene editing tools like CRISPR are advancing disease modeling.
  • CRISPR/Cas9 shows potential for treating genetic mutations.

Purpose of the Study:

  • To review CRISPR/Cas9 technology for retinal disease research.
  • To discuss applications of CRISPR/Cas9 in ophthalmology.
  • To explore CRISPR/Cas9's therapeutic potential.

Main Methods:

  • Review of CRISPR/Cas9 technology and its variants.
  • Comparison of Cas9 with other endonucleases.
  • Analysis of current research applications in retinal disease models.

Main Results:

  • CRISPR/Cas9 is a versatile tool for creating disease models.
  • CRISPR/Cas9 facilitates the study of genotype-phenotype correlations.
  • CRISPR/Cas9 enables genome editing for potential mutation correction.

Conclusions:

  • CRISPR/Cas9 is crucial for understanding retinal disease pathophysiology.
  • CRISPR/Cas9 holds promise for developing novel therapies.
  • CRISPR/Cas9 gene surgery could revolutionize retinal disease treatment.