article thumbnail

Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice

Broad Institute

In the new work published today in Nature Biotechnology , the team adapted engineered virus-like particles (eVLPs) that they had previously designed to carry base editors — another type of precision gene editor that makes single-letter changes in DNA.

article thumbnail

DNA Barcodes Could Streamline Search for New Drugs to Combat Cancer

NIH Director's Blog: Drug Discovery

Instead of the black, printed stripes of the Universal Product Codes (UPCs) that we see on everything from package deliveries to clothing tags, they used short, unique snippets of DNA to label cells. DNA barcoding has already empowered single-cell analysis, including for nerve cells in the brain.

DNA 52
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Epigenetic editing: the next generation of genetic medicine

Drug Target Review

The reasons for this are multifaceted, including concerns over the safety of directly altering DNA sequences and subsequent regulatory restrictions that have arisen as a result. The epigenome (meaning ‘above the genome’) is a system of reversible marks regulating how the DNA is read, translated and used. What is epigenetic editing?

DNA 105
article thumbnail

DNA Analysis Finds New Target for Diabetes Drugs

NIH Director's Blog: Drug Development

Credit: Jane Ades, National Human Genome Research Institute, NIH Type 2 diabetes (T2D) tends to run in families, and over the last five years the application of genomic technologies has led to discovery of more than 60 specific DNA variants that contribute to risk.

DNA 52
article thumbnail

Prime editing efficiently corrects cystic fibrosis mutation in human lung cells

Broad Institute

Next, researchers will need to develop ways to package and deliver the prime editing machinery to the airways in mice and ultimately humans. Prime editing, a more flexible and controlled kind of gene editing that does not require double-stranded breaks, could help address this limitation. The new approach also generated 3.5

article thumbnail

Viral Vectors 101: An Introduction to AAV

addgene Blog

Adeno-associated virus (AAV) is a single stranded, Parvoviridae DNA virus, packaged in a non-enveloped icosahedral capsid, that can be used to express genes of interest in cell and animal models. This post was contributed by guest blogger Didem Goz Ayturk with edits and updates from Addgenies Karen Guerin and Susanna Stroik.

Virus 67
article thumbnail

Unveiling neoantigen-directed cancer treatment

Drug Target Review

As part of the TCR discovery process and preclinical data package, Adaptive performs rigorous functional characterisation and safety assessments of potent, therapeutic grade antigen-specific TCRs. While tumour cells share a majority of their DNA with healthy cells, they also carry numerous unique mutations.

Treatment 105