Cures -not yet

The research into breast cancer discovered gene defects that caused the cancer. Ever since the research on how to edit genes has grown.

There is not a cure yet but our best hope is gene editing using Crispr. The following link is about CRISPR-based gene editor designed to target adenine ⎯ one of the four main DNA building blocks from Rice University

DNA is a molecule made up of four chemical bases: adenine (A), cytosine (C), guanine (G), and thymine (T). For the two strands of DNA to zip together, A pairs with T, and C pairs with G. Each pair comprises a rung in the spiral DNA ladder. The order of these building blocks in a DNA molecule determines the genetic sequence. These sequences make genes—the instructions for making specific proteins—and other genetic elements. When a mutation occurs, it means one of these bases have been replaced with a different one.

We do not have a cure yet but we are getting closer. Editing our defective genes is the best way to get there.

To get a better understanding of what happened to our DNA, take a look at the Cleveland Clinic's description.

A groundbreaking gene therapy developed at Nationwide Children’s Hospital to treat the most common form of muscular dystrophy has been approved by the FDA, bringing hope to thousands diagnosed with this devastating and life-threatening disease. Duchenne muscular dystrophy, or DMD, mostly affects boys. Symptoms begin with muscle weakness and mobility issues in childhood and progress to cardiovascular and respiratory issues in early adulthood. Watch the videos for more insight. Duchenne muscular dystrophy is inherited in an X-linked recessive pattern.

Research in Duchenne MD

The CRISPR/Cas9 method is based on bacteria’s natural system to protect itself from viral infections (similar to an immune system). When bacteria detect the presence of foreign (viral) DNA, the CRISPR proteins capture a piece of the virus’ DNA and insert that piece into the bacteria’s own genome. The bacteria then uses the “immunizing” viral DNA piece to produce “antibodies” that recognize and defend against future viral attacks.

Dominant mutations often have a “bad” or destructive piece of information that shouldn’t be there. AVVs can’t remove bad RNA. But CRISPR can. Like a tiny pair of scissors it can cut a bad piece out and insert a good piece in, repairing the gene and making it function properly.

This PDF article explains how they are using Crispr/Cas9 mRNA delivery to enhance muscle power.

FDA APPROVES 1ST Crispr treatment in the US. Casgevy, a cell-based gene therapy, is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises. Casgevy is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of genome editing technology.