Medical research

Discovery challenges the foundations of gene therapy

A new publication by scientists from Children's Medical Research Institute has challenged one of the foundations of the gene therapy field and will help to improve strategies for treating serious genetic disorders of the ...

Neuroscience

Getting gene therapy to the brain

A lone genetic mutation can cause a life-changing disorder with effects on multiple body systems. Lysosomal storage diseases, for example, of which there are dozens, arise due to single mutations that affect production of ...

Diseases, Conditions, Syndromes

Ethiopia must do more to stop recurring chikungunya outbreaks

Ethiopia's second largest city, Dire Dawa, is dealing with a chikungunya fever outbreak. Around 20,000 cases of the mosquito-borne viral infection have been reported since the outbreak started in March 2019. This is the second ...

Neuroscience

Economic, fast and efficient cell-type targeting

It is a remarkable proof for the concept of IOB: working closely hand in hand, our molecular and clinical researchers have developed a library of 230 adeno associated viral vectors (AAVs), each with a different synthetic ...

Medical research

Gene therapy cassettes improved for muscular dystrophy

Experimental gene therapy cassettes for Duchenne muscular dystrophy have been modified to deliver better performance. The cassettes, which carry the therapy into muscle cells, contain newer versions of a miniaturized treatment ...

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