CRISPR therapy for Thalassemia

Hi All,

Read the following today which has been described as a promising and revolutionary therapy for genetic diseases like sickle cell and Huntington.

http://www.independent.co.uk/news/science/crispr-gene-therapy-scientists-call-for-more-public-debate-around-breakthrough-technique-8927606.html

Is it possible to treat Thalassemia also using this method?

Regards,
Nijaguna

Eventually, this may have many uses, including for thalassemia but this is a very early step.

This therapy can be very promising for thalassemia, and so many other illnesses.  I hope that progress continues in the field of biotechnology at a rapid rate.  I have a feeling if people continue to elect governments that promote advancements in science and health we will have safer and more effective cures for all diseases in the near future. 

This is an update on this technology.

A GENETIC disease has been cured in living, adult animals for the first time using a revolutionary genome-editing technique that can make the smallest changes to the vast database of the DNA molecule with pinpoint accuracy.

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Scientists have used the genome-editing technology to cure adult laboratory mice of an inherited liver disease by correcting a single "letter" of the genetic alphabet which had been mutated in a vital gene involved in liver metabolism.

A similar mutation in the same gene causes the equivalent inherited liver disease in humans – and the successful repair of the genetic defect in laboratory mice raises hopes that the first clinical trials on patients could begin within a few years, scientists said.

The success is the latest achievement in the field of genome editing. This has been transformed by the discovery of Crispr, a technology that allows scientists to make almost any DNA changes at precisely defined points on the chromosomes of animals or plants.

Crispr, pronounced "crisper", was initially discovered in 1987 as an immune defence used by bacteria against invading viruses. Its powerful genome-editing potential in higher animals, including humans, was only fully realised in 2012 and 2013.

Since then, there has been an explosion of interest in the technology because it is such a simple method of changing the individual letters of the human genome – the 3 billion "base pairs" of the DNA molecule – with an accuracy equivalent to correcting a single misspelt word in a 23-volume encyclopaedia.

In the latest study, scientists at the Massachusetts Institute of Technology (MIT) used Crispr to locate and correct the single mutated DNA base pair in a liver gene known as LAH, which can lead to a fatal build-up of the amino acid tyrosine in humans and has to be treated with drugs and a special diet.

INJECTIONS

The researchers effectively cured mice suffering from the disease by altering the genetic make-up of about a third of their liver cells using the Crispr technique, which was delivered by high-pressure intravenous injections.

"We basically showed you could use the Crispr system in an animal to cure a genetic disease, and the one we picked was a disease in the liver which is very similar to one found in humans," said Professor Daniel Anderson of MIT, who led the study.

http://www.independent.ie/world-news/americas/hopes-for-human-trials-after-mice-cured-by-dna-editing-30205508.html

Hi all,

Please read the latest update today:

http://www.independent.co.uk/life-style/health-and-families/health-news/new-gene-correction-technique-could-revolutionise-treatment-9649983.html

Kind regards,
Alicia & Shastri

This is indeed interesting development. Thanks for posting this!!

Hope Scientist continue to work on this approach and find success soon...

(About the August article!)

Here is a topic on reddit with a few more useful comments: http://www.reddit.com/r/Futurology/comments/2cppiq/two_mutations_in_the_haemoglobin_gene_of_a/

The effective gene therapies we are likely to see applied in the clinic in the next 10-15 years will nearly all be related to blood (including immune) disorders, because circulating cells are easy to isolate, manipulate, and reintroduce.

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A blood transfusion would just be like giving him some healthy blood. To make the patient actually start producing healthy red blood cells on his own I'm thinking more along the lines of a bone marrow transplant.

I thought BMT has already been atleast an option to cure thalassemia for a long time. What's the difference with the new methods? That a patient does not need a donor anymore?

BMT has the risk of graft vs host disease, which does cause mortality in some patients. This risk is not present in gene therapy because you don't receive any donated genetic material.