news
New target in the fight against heart disease
US scientists have new insight into how heart cells enable unhealthy growth, and identify a new target to intervene against heart disease.
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Gene Therapy
Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
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Drug Target Review – Issue 4 2022
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.
news
Researchers have engineered AAV vectors that cross the blood-brain barrier
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.
news
Potential treatment for neurological disorders targets brain cells
UCL researchers have developed a possible new treatment for neurological and psychiatric diseases, that works by reducing the excitability of overactive brain cells.
news
New control system created for synthetic genes
MIT researchers have developed a technique that could help the production of monoclonal antibodies and other useful proteins.
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Decibel Therapeutics submits IND application for hearing loss gene therapy candidate
Decibel Therapeutics has submitted an IND application for a Phase I/II trial of DB-OTO, a gene therapy candidate to restore hearing.
webinar
Power up your LNP and AAV sample prep with Unagi
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.
news
Improved AAV could cross blood-brain barrier and deliver gene therapies to CNS
A newly developed AAV has shown promise at delivering gene therapies to the brain in mice and non-human primates.
whitepaper
Product hub: CD34+ haematopoietic stem cells: One cell type, multiple viable sources
Learn how you can mobilise CD34+ haematopoietic stem cells and how they can be isolated immediately after collection.
news
Researchers develop NPHP5 gene therapy for rare ciliopathy
The researchers found that augmenting NPHP5 gene rescues cilia defects in light-sensing cells derived from ciliopathy patients.
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Researchers find that CRISPR gene editing can give rise to cell toxicity and genomic instability
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.
podcasts
Episode 11 – CRISPR with Dr Jakob Haaber, SNIPR Biome & Dr Richard Fox, Infinome Biosciences
Join leading experts as they discuss the wide range of uses for CRISPR, including for therapeutics and biomanufacturing.
news
Gene therapy approach shows promise in treating ALS
In rodent models of ALS, the introduced neuroprotective protein slowed disease progression and increased life span.
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ELRIG: New modalities in pharmacology and drug discovery
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.
news
Concerns for future gene therapy testing in laboratory mice
Researchers have found that new sickle cell disease gene therapies depend on choosing the right laboratory mice.
