"This type of gene editing should also allow for the treatment of diseases whose origin is not genetic or whose origin cannot ...
One in every 10 people worldwide is impacted by a rare genetic disease but about 50% of them remain undiagnosed despite rapid ...
Get Instant Summarized Text (Gist) A new approach using personalized antisense oligonucleotides (ASOs) and patient-derived organoids offers promising treatment options for rare genetic diseases ...
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches -- gene therapy ...
Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as ...
AI-driven genomics startups leverage technology to analyze genetic data, predict predispositions, and offer personalized ...
The announcement was made at Arab Health 2025 where Sidra Medicine will highlight its international patient service programmes ...
Around 80% of rare diseases have a genetic cause, almost 70% of which present in childhood, about 95% lack approved treatments, and the average time for an accurate diagnosis is 4·8 years. In the past ...