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Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
A new Drug Target Review issue is now ready to download! This issue features articles which explore how artificial intelligence can enhance screening and ways to find new hits through simultaneous orthogonal screens. Also included are articles on CRISPR, immuno-oncology and RSV vaccines.
In primate models, researchers have successfully shown that developed AAVs can cross the blood-brain barrier, which keeps many drugs from getting into the brain.
UCL researchers have developed a possible new treatment for neurological and psychiatric diseases, that works by reducing the excitability of overactive brain cells.
MIT researchers have developed a technique that could help the production of monoclonal antibodies and other useful proteins.
Decibel Therapeutics has submitted an IND application for a Phase I/II trial of DB-OTO, a gene therapy candidate to restore hearing.
12 October 2022 | By Unchained Labs
Watch this on-demand webinar to see first-hand data results demonstrating the power and flexibility of Unagi for nucleic acids, LNPs and AAVs.
A newly developed AAV has shown promise at delivering gene therapies to the brain in mice and non-human primates.
Learn how you can mobilise CD34+ haematopoietic stem cells and how they can be isolated immediately after collection.
The researchers found that augmenting NPHP5 gene rescues cilia defects in light-sensing cells derived from ciliopathy patients.
Using computational methods, researchers analysed the most popular CRISPR library designed for human cells and found 3,300 targeted spots that show strong toxic effects.
Join leading experts as they discuss the wide range of uses for CRISPR, including for therapeutics and biomanufacturing.
In rodent models of ALS, the introduced neuroprotective protein slowed disease progression and increased life span.
Drug Target Review’s Ria Kakkad recently visited ELRIG and the British Pharmacological Society’s conference on New Modalities in Pharmacology and Drug Discovery which took place in London, UK. This article shares some of the key takeaways from the event.
Researchers have found that new sickle cell disease gene therapies depend on choosing the right laboratory mice.
Researchers have found that inherited mutations in the MINAR2 gene causes deafness. The findings suggest that deafness could be treated with genetic therapies.
