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
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
Campbell
Is the AD referred to above really AZ ? If not what is it ?
John, I also noticed AD in the text. It should have read AZ and is now corrected. thanks
Campbell, another very interesting blog. Well presented!
I have noticed what seems to be ever-increasing cases in AZ over the past 20+ yrs with my mother (now passed) and many of her peers, including her brother and sister, who also suffered from this distressing condition for thousands of families and friends before their passing. Both of my grandmothers however lived to ages in excess of 95 yrs with highly acute faculties. I was tending to beleive AZ was a genetic disease developed from changes to more modern lifestyles and variations to more global diets.
Hopefully CRISPR research and final application once proven, will be a godsend to those nearing the potential of developing AZ and provide hope for us and all our descendents.
Peter, thanks. Hopefully, the fix will not be too far away. In the meantime, I think the greatest defence is regular exercise. It staves off most diseases, e.g high blood pressure, type 2 daibetes, heart problems and most other contributing ails. Many other things, stopping smoking, obesity, moderate alcohol are avoidable. Boring? Not if you feel great and want to take on the world! Individual choice. The only things not controllable are genes but for the vast majority, this is not an issue. Maybe CRISPR can fix it.
Great article Campbell / one that is applicable to an old person like me. I am not sure what my genome is but I have seen known people waste away with AD.
Your advise Re exercise and moderation in food and drinks is appropriate in the circumstances.
An article that was very informative without any jargons.
Keep up the good work that greatly enlightens and benefits people like me – seniors.
Bala
Good to hear from you, but we both know you are not old! At least from my viewpoint! You can have your DNA profiled for $89 in Australia. Just Google it. I had mine done through Ancestry.com
Campbell
Excellent and manageable info. Thank you.
I will dig in a little further on this subject which seems to touch us all.
John, Let’s hope not all of us suffer from it, but certainly, we know people who do! The beauty about CRISPR is with time virtually all diseases, certainly hereditary ones may be eliminated. Not in our lifetime but it is an amazingly clever tech breakthrough.
Great article Campbell, enjoyed reading it, very informative.
Thanks, Frank – good to know
Stress management is important – and regular exercise (brain and body) is great medicine, along with daily laughter!
Yes Tony, in these times a good laugh keeps us sane!
Hi Campbell, Thanks so much for the informative material on dementia. My wife is struggling with aphasia which is a frontotemporal dementia. At this stage it is slow but it does make life difficult for her. Crispr is certainly an amazing breakthrough . Looking forward to reading your next article.
Cheers John
John, good to hear from you, but sorry to hear about the aphasia problem.