All gene therapy articles
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NewsBreakthrough gene therapy offers hope for rare, deadly heart disease in young men
Researchers have designed a gene-therapy strategy that could transform the treatment of arrhythmogenic right ventricular cardiomyopathy type 5 (ARVC5), a rare and deadly heritable disease that particularly affects young men.
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NewsEvaluating DNA impurities in recombinant adeno-associated virus
A new study indicates that DNA impurities derived from plasmid and host cell DNA are encapsulated into recombinant adeno-associated virus (rAAV) capsids as single-stranded DNA.
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NewsPrevalence of neutralizing antibodies to AAV2 and AAV9 in individuals with Niemann-Pick disease
A new study shows that more than half of individuals with Niemann-Pick disease, type C1 (NPC1) who were tested lacked neutralizing antibodies against either adeno-associated virus (AAV) 2 or AAV9.
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NewsScottish biotech company Lentitek secures £1m funding to advance next generation cancer treatments
Biotech company Lentitek Ltd has secured £700,000 in private funding from Equity Gap, bringing its total investment to £1 million in the last six months. It develops manufacturing technologies for lentiviral vectors, used with CAR-T cell and gene therapies.
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NewsGene-delivering viruses cross into brain in step toward gene therapy for neurological diseases
A family of adeno-associated viruses (AAVs) has been developed that are capable of delivering gene therapies to the brain, a particularly challenging target tissue.