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Showing posts with the label CRISPR- CAS9

Use CaRROT to turn on/off genes

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Genetic modification is a hugely debated subject as well as a highly interesting one. Being able to genetically modify a plant or an animal (humans included) is a huge achievement towards understanding our genes and their function. Over the past few years, CRISPR- Cas9 has been in the news for the simple reason that the technology allows labs to easily edit genomic sequences in live cells. The simplicity of the technique lies in the fact it requires a CRISPR associated protein 9 (Cas 9) and a single guide RNA (gRNA) to edit the region of your interest. 
The technology also brings with it the promise of curing genetic diseases like sickle cell anemia, beta-thalassemia and cystic fibrosis, among others. Many privately funded labs are already in the race to design and manufacture CRISPR-Cas9 derived therapeutics that could make genetic diseases a thing of the past.CRISPR-Cas9, however, has its limitations. For one, the editing that it can easily bring about, is not very specific,…

Future deafness treatments will be genetic in nature

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As per estimates released in 2015, Deafness or hearing loss affects 1.1 billion people globally. There are many causes of deafness ranging from trauma to the ear to severe infections, developmental issues as well as exposure to noise. However, 80% of deafness cases are non-syndromic i.e. do not show any other symptoms such as ear malformations and are also genetic in nature. 
Cochlear implants have met a lot of success in recent years but come with its own set of benefits and risks and are definitely not a permanent cure. Like with any other genetic condition, deafness, too, has been known to be non-curable. But recently published research puts hope on the horizon for patients with deafness that gene editing technology could one day become mainstream and allow treatment for genetic deafness. 
Gene Editing technologies have taken the scientific world by storm in the recent years. Among these, CRISPR - Cas 9 system has gained quite a reputation for itself. What was published as general…

The story of Ringo, a dog who escaped Duchenne Muscular Dsytrophy!

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By definition, a genetic disorder is one caused due to defects arising in genes. If you have inherited the defective gene or genes, then you are bound to be affected by the disorder.



A common example is Duchenne Muscular Dystrophy, caused in humans by a defective copy of the Dystrophin gene (DMD) that encodes the dystrohin protein. DMD gene is located on the X chromosome. Since, females in humans have two X chromosomes, one defective copy of the X chromosome makes the person a carrier for the disorder. Muscular Dystrophy usually does not affect females, unless she is the progeny of an affected father and carrier mother. However, males, who inherit only one X chromosome from their mother, have a 50% chance of getting the defective copy of the gene from a carrier mother and therefore are at 50% risk of being affected by muscular dystrophy. That's the thumb rule.

Meet Ringo, a male Golden Retriever, the exception!  


Golden Retrievers are also susceptible to muscular dystrophy, called th…