New Discovery May Help In Treating Hereditary Hearing Loss
There has been an exciting new discovery for anyone with hereditary hearing loss due to genetic mutation. Currently, cochlear implants are the most advanced intervention for children and adults with this type of hearing loss. The findings of this study could lead to a treatment where people are actually able to regenerate healthy genes to replace their mutated ones, using stem cells. This is a huge leap in audiology findings and could revolutionize the way doctors and scientists currently treat and view patients with hereditary hearing impairment.
The original article, published in Stem Cells Translational Medicine uses complicated and technical jargon to explain the process. We’ve decided to explain these findings in a way that is understandable and digestible – because they are quite exciting, and we want everyone to know about them.
The Technical Findings
Virtual-Strategy Magazine synthesized the study by making this statement in their recent article about the publication,
“The team began by generating three iPSC lines from the urinary cells of a 7-year-old hearing loss patient with compound MYO7A gene mutations, her asymptomatic father and a healthy 26-year-old female donor, respectively. The iPSC line from the 7-year-old hearing loss patient were used to correct the MYO7A mutation with CRISPR/Cas9 technique, resulting in morphologic and functional recovery of hair cell-like cells derived from the corrected iPSCs.
“The current findings confirmed our hypothesis and might provide further insight into what is behind sensorineural hearing loss. Our hope is this will facilitate the development of iPSC-based gene therapy for genetic disorders,” Dr. Min-Xin Guan, another lead investigator, concluded” (Durham, 2016).
Whoa, What Does that Mean? First, let’s define the terms…
Hair Cells – What? We hear with our hair? No. But that would be pretty cool, right? When talking about hearing, “hair cells” are the sensory receptors located in the cochlea of our inner ear. They detect sound through movement, and convert this information into sounds our brain can hear and understand. Genetic mutations in these hair cells cause one type of hereditary hearing loss.
Induced Pluripotency Stem Cells (iPSCs) – Derived from the latin terms pluri meaning many, and potency meaning power, these are adult stem cells that have the ability to differentiate into many different types of cells that have different functions in the body. Induced pluripotency stem cells have been put through a process to allow them to become plutipotent, even if they were not beforehand.
CRISPR/Cas9 technique – This is a complicated and difficult gene editing technique that was invented by a group of scientists from the University of Berkeley in 2012. This technique can target and modify DNA with astonishing accuracy, and has gained considerable excitement amongst the scientific community in recent years.
Now, Let’s Try That Again…
A complicated gene editing technique was used to help a 7-year-old girl with hereditary hearing loss. The scientists used genes from the child’s father, who does not have hearing loss and a female donor. Genes from the donors urinary tracts had been put through the process to allow them to become induced pluripotency stem cells (iPSCs), which are cells that are able to morph into multiple types of cells. These iPSCs were taken from the two donors and compounded with the child’s genes, using the gene editing technique called CRISPR/Cas9. The results were that the child was able to develop recovery of cells that were similar to hair-cells – and they were functional. Lead investigators on this study are excited about the result, and hope it can develop into a new treatment and therapy for others with hereditary hearing loss due to gene mutations.
To read the Virtual-Strategy Magazine article quoted in this blog, click here.
To read the full study from Stem Cell Translational Medicine, click here.