CRISPR to Cure Alzheimers?

What is CRISPR? 

4 scientists developed CRISPR to edit DNA in 2012. This was recognised as the breakthrough of the year in 2015. Its first application is to eliminate hereditary diseases, either in a person’s body and/or eliminating it from all future children. After that, it may be used to eliminate most diseases. It has been constantly improved, the latest version is CRISPR Cas 9.

Definitions

RNA is a single helix, DNA double

The Following is Brief.

• Genes: (part of DNA) Every person has two copies of each gene, one from each parent.
• DNA (an acronym) is a molecule in all the cells of living organisms that transfers information to consecutive generations.
• RNA is like a reader that decodes DNA, e.g., mRNA (messenger RNA) tells vaccines what to do, as in Pfizer and Moderna.
• Your Genome is your complete genetic information, all your DNA, RNA, etc.
• The above helixes (or helices!) are in every cell of the body. The human body has trillions of cells. 
• CRISPR Cas 9 sends in RNA to edit the DNA in every cell to fix faults

CRISPR Successes in Trials on Humans

• Sickle Cell Anemia: Red blood cells become shaped like sickles and don’t move easily through the bloodstream creating a lack of oxygen
• Hereditary Blindness
• Beta Thalassemia. Causes a lack of oxygen to parts of the body, is a version of sickle cell anemia
• Transthyretin Amyloidosis: Symptoms are shortness of breath, fatigue and swelling of the lower legs. This is the first application where the therapy gene was inserted directly into the bloodstream. This may be the future of getting to the faulty DNA
• Cancer: Effective in curing certain cancers
• Brain Disorders: It is only a matter of time before CRISPR trials get approved for brain disorders

Alzheimer’s – referred to as ‘AZ’ below

What is it?

Normally associated with aging, AZ constitutes 60% to 70% of all causes of dementia, which is the general term for brain disorders. If you want detailed information click here and here. At present, there is no cure. Other forms of dementia are vascular dementia, frontotemporal dementia and Lewy body disease.

At present, there are about 50 million people with dementia worldwide and this is predicted to treble by 2050 unless a cure is found. Head trauma, concussion, etc. are also causes of dementia, but may not show up until later in life.

Background:

• When AZ occurs, the experts refer to the build up of amyloid plaques (like cholesterol build up in arteries) and ‘entanglements’ in the brain
• There are certain genes (APOE4 is the main one) that do affect AZ onset and in particular early onset if both parents had AZ and you have 2 or more of APOE4. However don’t despair, the chances are low
• Women in their 60s are twice as likely to develop AZ as men and research is trying to determine why. See here. However, statistically, women live longer than men
• Having one or two APOE4 genes increases the risk of developing Alzheimer’s. About 25 per cent of people carry one copy of APOE4, and 2 to 3 per cent carry two copies. But there are many ways to reduce the likelihood – see below
• What is good for the heart is good for the brain. High blood pressure and lack of exercise are the 2 main factors increasing the chances of AZ
• Ways to love your brain – click here

CRISPR Editing Results So Far

Cas 9 is the Latest Version of CRISPR

It is early days for CRISPR as most of the research is still being done on animals or in Petri dishes, but there is promise – see examples below!

In the largest biology project ever, the NIH (US National Institute of Health) is uniting Alzheimer’s and stem cell researchers to see if CRISPR can defeat this awful disease. 

In Canada, researchers have identified a genetic variant that reduces AZ likelihood by a factor of four. A CRISPR edit may be able to produce it

One of the first functions to be affected in AZ is short term memory. When CRISPR-Cas9 was used to correct the gene causing this, the scientists were able to restore correct function.

In another study, scientists used CRISPR to specifically target and disrupt the mutation of what they refer to as the AAP gene. This could be a potential treatment.

Please comment below if you need more detailed explanation.

References

• Syntghego
• Dementia Australia
• Medic Australia
• Alzheimer’s Association
• Healthline
• WHO – Dementia

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