Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Site-Targeted Drug Delivery Systems: Polymeric Carriers01:24

Site-Targeted Drug Delivery Systems: Polymeric Carriers

Polymeric carriers enhance targeted drug delivery by increasing efficacy while minimizing off-target effects. These carriers comprise a biodegradable polymeric backbone integrated with functional elements that enable targeting, improve physicochemical properties, and regulate drug release.Targeting MechanismsThe targeting ability of polymeric carriers is mediated by a homing device, which is a molecular recognition component designed to selectively bind to specific tissues or cells. Monoclonal...
Conservative Site-specific Recombination and Phase Variation02:53

Conservative Site-specific Recombination and Phase Variation

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...
CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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...
Modified-Release Drug Delivery Systems: Site-Targeted01:24

Modified-Release Drug Delivery Systems: Site-Targeted

Site-targeted drug delivery systems enhance therapeutic efficacy while minimizing systemic toxicity and treatment costs. Unlike conventional methods, these systems ensure precise drug delivery, improving bioavailability and reducing side effects. Targeted drug delivery is classified into three levels. First-order targeting directs drugs to the capillary beds of specific organs or tissues. Second-order targets specific cell types, such as tumor cells, using receptor-mediated interactions.
CRISPR01:59

CRISPR

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 Short...

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Real-time single-particle imaging of functional lungs reveals mosaic-like patterns of aerosol deposition in alveoli.

Nature biomedical engineering·2026
Same author

Investigating the role of mimosine-induced genotoxic stress through DNA repair profiling.

Molecular therapy. Nucleic acids·2026
Same author

Endothelial MHC expression is required to initiate T cell-mediated rejection of 3D-printed skin grafts.

JCI insight·2026
Same author

Advances in peptide nucleic acid for targeting RNA and genomic DNA.

Cell reports. Physical science·2026
Same author

Prenatal Intraportal Delivery of Polymeric Nanoparticles to Fetal Rhesus Monkeys (<i>Macaca mulatta</i>).

Human gene therapy·2026
Same author

Combining anti-gene γPNA with small molecules and RNA inhibitors: A strategy to enhance anti-tumor efficacy.

Molecular therapy. Nucleic acids·2026

Related Experiment Video

Updated: Jun 1, 2026

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
09:51

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

Polymer delivery systems for site-specific genome editing.

Nicole Ali McNeer1, Erica B Schleifman, Peter M Glazer

  • 1Department of Biomedical Engineering, Yale University, New Haven, CT 06511, USA.

Journal of Controlled Release : Official Journal of the Controlled Release Society
|May 31, 2011
PubMed
Summary

Peptide nucleic acids enable precise gene editing in blood stem cells for inherited disorders and HIV. Biodegradable nanoparticles offer a promising delivery method for this advanced gene therapy.

More Related Videos

Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation
09:51

Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation

Published on: February 2, 2016

A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells
10:07

A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells

Published on: August 25, 2017

Related Experiment Videos

Last Updated: Jun 1, 2026

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
09:51

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation
09:51

Establishment of Genome-edited Human Pluripotent Stem Cell Lines: From Targeting to Isolation

Published on: February 2, 2016

A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells
10:07

A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells

Published on: August 25, 2017

Area of Science:

  • Molecular Biology
  • Gene Therapy
  • Nanotechnology

Background:

  • Peptide nucleic acids (PNAs) facilitate site-specific DNA recombination for genetic modification.
  • Hematopoietic stem and progenitor cells (HSPCs) are targets for treating blood disorders and HIV.

Purpose of the Study:

  • To review the application of engineered nanoparticles for gene editing in human hematopoietic cells.
  • To address challenges in delivering gene editing tools to hematolymphoid cells.

Main Methods:

  • Utilizing triplex-forming PNAs for targeted DNA integration.
  • Employing biodegradable polymer nanoparticles for intracellular delivery.

Main Results:

  • Demonstrated potential for site-specific correction of genetic mutations in HSPCs.
  • Highlighted the possibility of modifying CCR5 to combat HIV infection.

Conclusions:

  • Engineered nanoparticles show promise for efficient genome editing in hematopoietic cells.
  • This approach could advance ex vivo and in vivo gene therapy for genetic diseases and viral infections.