news
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.
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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.
news
P53 could be key to therapies for salivary gland cancer
Mouse models show that activating a non-mutated form of the gene could lead to developing therapies for salivary gland cancer.
news
CAR-T therapy becomes a powerful tool against an autoimmune disease like MS
US researchers have shown that CAR-T therapy can be used to eliminate unwanted cells that cause autoimmunity.
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Drug Target Review Cell and Gene Therapy Supplement 2022
This supplement focuses on how cell and gene therapy can target diseases such as cancer and reduce elevated lipoprotein(a).
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CIRM grant $4.8m to fund gene therapy for rare disease
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
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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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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.
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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.
