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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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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

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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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Related Experiment Video

Updated: Jul 2, 2025

Genome Engineering of Primary Human B Cells Using CRISPR/Cas9
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Cyclization-enhanced poly(β-amino ester)s vectors for efficient CRISPR gene editing therapy.

Xianqing Wang1, Yinghao Li1, Sigen A2

  • 1Charles Institute of Dermatology, School of Medicine, University College Dublin, D04 V1W8 Dublin, Ireland.

Journal of Controlled Release : Official Journal of the Controlled Release Society
|February 24, 2024
PubMed
Summary

New cyclic poly(β-amino ester)s (CPAEs) show enhanced gene delivery. Macrocyclic CPAEs improve DNA uptake and gene expression, demonstrating potential for gene editing in recessive dystrophic epidermolysis bullosa.

Keywords:
CRISPRCyclic architectureMacro ringNon-viral gene editingPoly(β-amino ester)s

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Designing, Packaging, and Delivery of High Titer CRISPR Retro and Lentiviruses via Stereotaxic Injection
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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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Area of Science:

  • Biomaterials Science
  • Gene Therapy
  • Polymer Chemistry

Background:

  • Poly(β-amino ester)s (PAEs) are versatile non-viral gene delivery vectors with evolving structures.
  • Highly branched PAEs (HPAEs) have demonstrated enhanced gene delivery efficacy.
  • A need exists for novel PAE architectures with improved transfection capabilities.

Purpose of the Study:

  • To introduce and characterize a new class of cyclic PAEs (CPAEs) for gene delivery.
  • To evaluate the gene transfection performance of CPAEs compared to HPAEs.
  • To assess the potential of optimized CPAEs for CRISPR-Cas9 gene editing in patient-derived cells.

Main Methods:

  • Synthesis of cyclic PAEs (CPAEs) using an A2 + B4 + C2 cyclization strategy.
  • Characterization of CPAE structures using 2D NMR and photoluminescence.
  • In vitro evaluation of DNA delivery, intracellular uptake, gene expression, and biocompatibility.
  • Application of MCPAEs for delivering CRISPR-Cas9 plasmid for gene editing in RDEB keratinocytes.

Main Results:

  • Two sets of CPAEs with distinct ring sizes and topologies were synthesized and confirmed.
  • Macrocyclic CPAEs (MCPAEs) exhibited significantly enhanced DNA intracellular uptake and gene expression compared to HPAEs.
  • MCPAEs demonstrated excellent biocompatibility.
  • MCPAEs successfully delivered CRISPR-Cas9 plasmids into RDEB patient-derived keratinocytes, achieving targeted gene editing.

Conclusions:

  • Cyclic PAEs, particularly MCPAEs, represent a promising advancement in non-viral gene delivery vectors.
  • MCPAEs offer superior gene transfection performance and biocompatibility.
  • MCPAEs show significant potential for therapeutic gene editing applications, including in genetic disorders like RDEB.