CRISPR is a gene-editing technique. This involves treating this inherited form of blindness by replacing the defective gene.
Patients are being enlisted to be a part of the study to test a gene-editing technique, CRISPR. It may be used to cure an inherited form of blindness.
Gene Therapy for Macular Degeneration
Gene therapy gives new hope for age-related blindness. Scientists were able to deliver the gene for a retinal opsin into the genome of ganglion cells using an adeno-associated viral vector. The blind ganglion cells were restored and became sensitive to light.
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‘CRISPR uses a tool that cuts or "edits" DNA in a specific spot, This technique targets to cater the kids and adults with a healthy version of the gene they require. It's planned to be a one-time treatment that permanently changes the person's native DNA.’
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People with inherited blindness mostly have healthy eyes but require a gene that converts light into signals to the brain so that it can interpret and enable sight.
The objective experimental treatment aims to supply kids and adults with a healthy version of the gene they lack, utilizing a tool that cuts or "edits" DNA in specific places. It would be a one-time treatment providing a permanent solution to the problem.
![Inborn Errors of Metabolism]( "Inborn Errors of Metabolism")
Inborn errors of metabolism are genetic disorders that hamper the body's metabolism resulting in severe clinical manifestations.
This fall, Editas Medicine and Allergan, the two companies will test this technique in up to 18 people around the United States, including Massachusetts Eye and Ear in Boston, starting .
This gene editing in people after birth is very different from the controversial work a Chinese scientist did last year —which was changing the DNA of embryos at the time conception in such a way that they can pass on the changes to future generations,but this technique aims not to affect the progeny in any way genetically.
Sangamo Therapeutics is the company which has tried to treat metabolic diseases by gene editing inside the body, using a tool called zinc fingers.
Potential and risks with CRISPR Scientists have found CRISPR to be very promising because it's a very simple way to do gene editing. Hence, researchers believe that it has great potential to cure or treat many diseases caused by gene flaws. However, it's so new that its risks are not fully known.
Source: Medindia
Inborn Errors of Metabolism
Inborn errors of metabolism are genetic disorders that hamper the body's metabolism resulting in severe clinical manifestations.
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This fall, Editas Medicine and Allergan, the two companies will test this technique in up to 18 people around the United States, including Massachusetts Eye and Ear in Boston, starting .
This gene editing in people after birth is very different from the controversial work a Chinese scientist did last year —which was changing the DNA of embryos at the time conception in such a way that they can pass on the changes to future generations,but this technique aims not to affect the progeny in any way genetically.
Sangamo Therapeutics is the company which has tried to treat metabolic diseases by gene editing inside the body, using a tool called zinc fingers.
Potential and risks with CRISPR Scientists have found CRISPR to be very promising because it's a very simple way to do gene editing. Hence, researchers believe that it has great potential to cure or treat many diseases caused by gene flaws. However, it's so new that its risks are not fully known.
Source: Medindia
New Gene Therapy Cures Babies With Bubble Boy Disease
Infants with X-linked severe combined immunodeficiency have fully functioning immune systems following treatment with gene therapy developed and produced at St. Jude Children's Research Hospital.
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