"This type of gene editing should also allow for the treatment of diseases whose origin is not genetic or whose origin cannot ...

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches -- gene therapy ...

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 ...

A new study shows that long-read sequencing has the potential to improve the rate of diagnosis while reducing the time to diagnosis from years to days -- in a single test and at a much lower cost.

After more than a year the HSHS Hospital/Prevea Clinic closures were announced, a couple of Eau Claire County boys have ...

A teenager diagnosed with a rare genetic eye disease which means she will lose her eyesight is desperate to see the world ...

The early stage company is developing a new approach to gene editing to treat rare genetic diseases. It does this using mRNA, the technology underlying the Covid vaccines, so the fact that Zaks is ...

Citation: Long read sequencing reveals more genetic information while cutting time and cost of rare disease diagnoses (2025, January 24) retrieved 2 February 2025 from https://medicalxpress.com ...

One in every 10 people worldwide is impacted by a rare genetic disease but about 50% of them remain undiagnosed despite rapid increases in genetic technology and testing. Even when a person does have ...

Ultragenyx Pharmaceutical is a mid-cap biotech firm with a promising pipeline and an optimistic outlook from analysts. Click ...

Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as ...