Misfolding proteins bring caution for gene therapies for haemophilia
US researchers have discovered a link between protein misfolding and liver cancer, that could help improve gene therapy for haemophilia.
List view / Grid view
Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
US researchers have discovered a link between protein misfolding and liver cancer, that could help improve gene therapy for haemophilia.
Mouse models show that activating a non-mutated form of the gene could lead to developing therapies for salivary gland cancer.
US researchers have shown that CAR-T therapy can be used to eliminate unwanted cells that cause autoimmunity.
This supplement focuses on how cell and gene therapy can target diseases such as cancer and reduce elevated lipoprotein(a).
CIRM grant will fund novel gene therapy that aims for single lifelong treatment of Friedreich’s ataxia, a progressive neuromuscular disorder; a second CIRM grant will advance efforts to leverage UC San Diego research on another rare disease
US scientists have new insight into how heart cells enable unhealthy growth, and identify a new target to intervene against heart disease.
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.