Deaf girl is cured in world first gene therapy trial (independent.co.uk)

Article summary

An 18-month-old British girl, Opal Sandy, has undergone a groundbreaking gene therapy trial to treat her auditory neuropathy, a condition that caused her to be born completely deaf. The trial, which used a gene therapy from biotech firm Regeneron, has shown promising results, with Opal's hearing now almost normal. The treatment involves delivering a working copy of the OTOF gene to the ear, which enables cells to communicate with the hearing nerve. This is the first time this type of gene therapy has been used to treat auditory neuropathy.

Main themes

• Gene therapy
• Auditory neuropathy
• Deafness treatment
• Medical breakthroughs
• Color blindness
• FDA regulations

What commenters say

• The article's gene therapy treatment is not based on CRISPR, but rather uses an adeno-associated virus to deliver a working copy of the OTOF gene to the ear.
• Some commenters believe that gene therapy could potentially be used to cure color blindness, but the FDA has decided that the risks are not worth the benefits for this condition.
• Others argue that individuals should be allowed to try experimental gene therapies, such as those for color blindness, as long as they provide informed consent and pay for the treatment themselves.
• There is disagreement over the effectiveness of color blindness glasses, such as EnChroma, with some claiming they are helpful and others arguing that they do not work as advertised.
• Some commenters think that the brain's ability to adapt to new sensory information, such as restored hearing or vision, is an important factor to consider when evaluating the potential benefits of gene therapy.
• The use of gene therapy to treat non-life-threatening conditions, such as color blindness, raises questions about the balance between risk and benefit, and who should be allowed to make decisions about experimental treatments.