Ethical Treatment Cure for Sickle Cell Disease

Contrary to the last International Human Genome Editing Symposium, the focus was Somatic Human Gene Editing (SHGE), wherein the non-reproductive cells of adults/children were utilized to explore cures for diseases. This approach makes changes in the patient’s DNA without causing those changes to be inherited by future offspring. In gene editing, there is always a possibility of unexpected, off-target mutations occurring, with unknown detrimental outcomes. While the human subject of a clinical trial should have …

CRISPR Causes Mutations that are Passed on to Offspring

It is 2022, and even with the explosion of medical research over the last decade using CRISPR gene editing, the unwanted and off-target mutations caused by the game-changing tool have not been controlled. Furthermore, studies consistently find that these mutations are directly inherited by any offspring produced. Indeed, CRISPR has facilitated great strides in curing single-genome diseases, such as sickle-cell anemia. But these are cases where adults were treated, and embryos/germ cells (eggs and sperm) …

Gene Edited Pig Heart Successfully Transplanted

In a ground-breaking first, researchers and surgeons at the University of Maryland Medical Center have successfully transplanted a gene-edited pig heart into a dying man. For the heart to not be rejected once transplanted, 10 separate genes were deleted or added using CRISPR, earning the donor animal the nickname of a “10 gene pig”. Over 100,000 patients are awaiting an organ transplant at any given time, and about 20 of them die each day. This …

2020 Was a Breakout Year for Crispr

NCER Comments:  Sickle Cell Anemia is a heritable disease that 1 in every 13 Black or African American babies possess when born. Red blood cells are abnormally shaped, so are unable to carry oxygen needed for all tissues and organs to survive. The average lifespan is only 40 years, but a new development using the CRISPR gene editing tool looks like a true cure.

WIRED

SCIENCE

2020 Was a Breakout

In It’s First Tough Test, CRISPR Base Editing Slashes Cholesterol Levels in Monkeys

NCER Comments:

The vast majority of adults are taking a daily Statin to manage their “bad” LDL and Triglyceride Cholesterol count, and many experience muscle pain as well as other adverse side effects. Using CRISPR gene editing, two companies, Verve and Beam Therapeutics, are intensely working on an ethical solution to high cholesterol which causes cardiovascular disease. Read the following to see the results.

 

As seen in

STAT

By SHARON BEGLEY

JUNE 27, 2020

A …

Diabetes Reversed in Mice with Genetically Edited Stem Cells Derived from Patients

NCER Comments:

Diabetes Mellitus (DM) affects more than 100 million adults in the US; that’s just under 10% of the population. Diabetes is associated with a 2x increase of death due to cardiovascular disease; and on average, patients with diabetes lose 6 years of their lifespan compared to those who do not. Additionally, DM is the leading cause of kidney failure, retinopathy and neuropathic pain. Can CRISPR-cas 9 be utilized to ethically cure …

He Jiankui faces three years in prison for CRISPR babies

MIT Technology Review

The Chinese scientist and two associates were sentenced after a secret trial.

Dec 30, 2019

The Chinese scientist who created the world’s first gene-edited children has been sentenced to three years in prison by a Chinese court.

He Jiankui, a biophysicist trained at Rice University and Stanford, shocked the world last year with his claim to have created genetically modified humans, twins referred to as Lulu and Nana. 

In

The newest gene editor radically improves on CRISPR

NCER Notes:

Research has shown that the CRISPR gene editing tool (which cuts out and replaces diseased DNA) holds great potential for healing disease, but currently poses a significant risk to humans due to frequent unintended mistakes, or “off-target” mutations in DNA. A solution is being pursued, but in the meantime, a new kind of gene editing, called “Prime Editing” has just been announced this week. Prime Editing uses much of CRISPR technology, but appears …

HIV Eliminated from the Genomes of Living Animals

NCER Notes:

With more than 36 M HIV infected people worldwide, and over 1 M living in the U.S., a cure for the disease is fervently sought. Researchers at our own UNMC in Omaha, Ne. believe they have found that cure! Mice were injected with human stem cells from cord blood in order to create a more human-like immune system. These mice were then infected with the HIV virus, and eventually treated with a long …

CRISPR Babies: When Will the World Be Ready?

NCER Notes:

As an alarming example of how the gene editing of embryos can be dangerous, the first gene edited babies born in China last year now have a high probability of a significantly shorter lifespan due to unexpected consequences of the editing. Because of this outcome, the World Health Organization has asked all countries to stop any further research leading to gene edited babies. In spite of these warnings, a Russian biologist plans to …