BioTech Firms Enthusiastic About Gene Editing Potential To Cure Disease

This announcement is exciting and inspiring. Without guidelines, what prevents use of gene editing on persons, diagnosed as having a disease, which may change brain functions? Psychiatry has a benevolent image, which allows corrupt doctors working for politicians or businessmen to designate a law abiding person insane and taking his freedom, forcing drugs on him. What barrier is there to prevent abuses using gene editing? Has technology outrun human understanding and morality? It is now two or three generations ahead of the law, which is useless controlling technological abuses available now to criminals and corrupt officials. The evolved politicians remain focused on getting re-elected and not offending anyone. 


Editing the brain

[From article]
The research being done at the Stanford School of Medicine, led by Dr. Matthew Porteus, is part of an accelerating research movement made possible using the new technique to try to cure genetic diseases such as sickle cell anemia and muscular dystrophy. These labs are steadily advancing through cell-based and animal trials, as fledgling biotech companies raise large sums of money needed to bring the therapies to market.
"Now, with lots of people -- hundreds or thousands of labs -- working with CRISPR, this means the possibility of actually finding a way to cure patients of disease increases dramatically," said Porteus, an associate professor of pediatrics and a pioneer in gene editing.
Using the campus's first-ever cell manufacturing plant, to be completed this spring, the Stanford team aims to start human trials in 2018. The researchers are targeting two severe blood diseases -- sickle cell anemia and beta thalassemia -- and several diseases that ravage the immune system.
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CRISPR has been in the crosshairs of controversy because of its profound potential to rearrange the basic building blocks of life. In December, experts gathered in Washington, D.C., to urge limits to its use in creating dangerous new organisms or "designer babies."
[. . .]
CRISPR -- which stands for "clustered regularly interspaced short palindromic repeats," or clusters of brief DNA sequences that read similarly forward and backward -- is the game changer. Only 3 years old, it works like the search-and-replace function of a computer.
A Palo Alto native who was educated at Harvard and Stanford, Porteus, 51, has spent his lifetime watching helplessly as patients declined from blood and immune system diseases such as sickle cell anemia, beta thalassemia and severe combined immunodeficiency disease.
These are very different diseases, he said. But they have this in common: All are caused by a single mutation in a single gene of a single cell type.
More than 10,000 human diseases are caused by a single gene defect, according to Dr. Maria Grazia Roncarolo, co-director of Stanford's Institute for Stem Cell Biology and Regenerative Medicine.
[. . .]


Snoop, snoop.

In November, the Cambridge, Mass., biotech startup Editas Medicine, co-founded by the Massachusetts Institute of Technology's Dr. Feng Zhang, announced that it planned to test a CRISPR-based gene therapy technique in hopes of curing a rare form of blindness by fixing a gene that controls the eye's photoreceptor cells. It has raised $120 million from investors, including Bill Gates and Google Ventures, on top of $43 million raised in 2013.
In December, the biotech firm CRISPR Therapeutics of Cambridge received an eye-popping $335 million investment from German pharmaceutical giant Bayer AG to form a joint venture that will research CRISPR-based treatments.
More sobering are the challenges posed by many of the world's more common diseases, such as autism, diabetes, heart disease or cancer, which involve multiple genes. And diseases that involve hard-to-reach tissues, such as Alzheimer's, are also daunting.

https://www.arcamax.com/currentnews/newsheadlines/s-1783024

JANUARY 10, 2016 12:00 AM
Researchers bring gene editing to patients with deadly diseases
BY LISA M. KRIEGER
San Jose Mercury News