Headline, January 01 2022/ ''' '' DNA -STUDENTS- DOT '' ''' : RESEARCH


 DOT '' ''' : RESEARCH

WE CAN EDIT A { PERSON'S - STUDENTS' DNA }. So why don't we? Revolutionized medicine may be at hand, but terrible barriers remain.

THE PARENTS OF A 2-YEAR-OLD GIRL write that their daughter ''could die within the next year'' because a genetic mutation is causing her heart to fail.

''TIME IS QUICKLY RUNNING OUT FOR ME,'' writes a man in his mid-30s whose DNA harbors a genetic mistake certain to destroy his brain within a matter of years.

''Watching my sons disintegrate before my eyes is heartbreaking,'' writes a mother of two children affected by a faulty gene that affects cognition, speech and mobility. One of her sons, she writes, is still walking and in college, but ''it is only a matter of time before he will be in a wheelchair and his cognition will decline.''

STORIES of human tragedies like these arrive in my inbox with increasing painful regularity.People write to see if I can build a medication to fix their genes and stave off an early, imminent death. Their wish is not futuristic : Many scientists, including me, build DNA fixes for a living.

OVER THE PAST DECADE - THOUSANDS of people have agreed to be genetically engineered in experimental trials to develop these treatments and to save lives.

Famously proposed 50 years ago, such fixes, or gene therapies, began earnest development in 1989. After fits and starts, the first real cures for children born with no functioning immune system arrived in the early 2000s.

Several approved gene therapy medicines now exist. All involve taking a virus, replacing its harmful contents with a disease-treating gene, and injecting it into a person [or exposing the person's cells to that virus in a dish and putting them back].

Though effective, these treatments remain cumbersome to build and jaw-droppingly expensive : One recently approved gene therapy for people with an inherited bleeding disorder costs a record-breaking $3.5 million for a single-use vial, making it the most expensive drug in the world.

Gene editing is much newer technology and builds on the gains of gene therapy. Instead of using a virus, however, gene editing relies on a molecular machine called CRISPR, which can be instructed to repair a mutation in a gene in nearly any organism, right that ''typo'' occurs.

Impressively versatile, potential applications for CRISPR range from basic science to agriculture and climate change.

In medicine, CRISPR gene editing allows physicians to directly fix typos in the patients' DNA. And so much substantive progress has been made in the field of genetic medicine that it's clear scientists have now delivered on a remarkable dream : word-processor-like control over DNA.

The first person to be gene-edited with CRISPR was treated only three years ago for a disorder of red blood cell production, and since then, the technology has been used to treat congenital blindness, sickle cell disease, heart disease, heart disease, nerve disease, cancer and H.I.V. 

While not all disease, have a single-gene base, most have a genetic component. Early studies suggest that conditions like heart disease, chronic pain and Alzheimer's disease could all be treated with CRISPR.

Dr. Jennifer Doudna, a winner of the 2020 Nobel Prize in Chemistry for CRISPR gene editing along with Emmanuelle Charpentier, aptly described it as a ''profound opportunity to change health care for many people.''

Scientists like me can now visualize an ideal scenario for the future of CRISPR medicines : When a 3-month starts to develop antibiotic-resistant infections, her primary care doctor orders a DNA test, and 48-hours later, the faulty gene that is preventing the development of normal immune system is identified.

''Not a problem. We will refer your child for corrective CRISPR therapy,'' says the physician to the devastated parents. ''The treatment will be covered by insurance and take all of two months.''

The Honour and Serving of the Latest Global Operational Research on Revolutionized Medicine, continues. The World Students Society thanks author Professor Fyodor Urnov, of molecular and cell biology at the University of California, Berkeley.

With respectful dedication to the Scientists, Patients in Need, and then Mankind, Students, Professors and Teachers of the world. See Ya all prepare and register for Great Global Elections on The World Students Society - the exclusive ownership of every student in the world : wssciw.blogspot.com and Twitter - !E-WOW! The Ecosystem 2011 :

Good Night and God Bless

SAM Daily Times - the Voice of the Voiceless


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