Gene Therapy will likely be the ultimate cure for SUB-PAR FMO3 function ('TMAU1').
The 1st 2 cases of CRISPR Gene Therapy in the USA are happening this year.
An update this week said they were going well (after initial therapy).
The 1st 2 cases are for Genetic blood disorders.
It's likely over the years it will be good for all genetic disorders (especially single gene).
Gene Therapy quick unchecked history
pre-2012 : very difficult to do
2012 : CRISPR method makes gene knock in/out very easy and affordable. Seen as a 'T-model Ford' moment.
2019 : Base Editing method shown. Most single-gene disorders will need base editing.
2019 : First 2 cases of use in humans for single-gene disorder.
FMO3 Gene Therapy
It's unknown when FMO3 would be trialed by a health system or private company.
All the 'obvious serious' genetic disorders are ;likely to be at the front of the queue.
They still need to see how the 2 cases get on (but looking good after around 3 months).
They will need to find out delivery methods, and spend millions on research etc.
But an excellent start, and FMO3 should (in most cases) be straightforward base editing (not always).
Link (New Atlas) : Encouraging early results from first human CRISPR gene therapy trials
NIH Directors Blog post
"As groundbreaking as CRISPR/Cas9 has been for editing specific genes, the system has its limitations. The initial version is best suited for making a double-stranded break in DNA, followed by error-prone repair. The outcome is generally to knock out the target. That’s great if eliminating the target is the desired goal. But what if the goal is to fix a mutation by editing it back to the normal sequence?
The new prime editing system, which was described recently by NIH-funded researchers in the journal Nature, is revolutionary because it offers much greater control for making a wide range of precisely targeted edits to the DNA code, which consists of the four “letters” (actually chemical bases) A, C, G, and T "
Link
CRISPR Therapeutics (done the 2 cases) : Press Release Link
The 1st 2 cases of CRISPR Gene Therapy in the USA are happening this year.
An update this week said they were going well (after initial therapy).
The 1st 2 cases are for Genetic blood disorders.
It's likely over the years it will be good for all genetic disorders (especially single gene).
Gene Therapy quick unchecked history
pre-2012 : very difficult to do
2012 : CRISPR method makes gene knock in/out very easy and affordable. Seen as a 'T-model Ford' moment.
2019 : Base Editing method shown. Most single-gene disorders will need base editing.
2019 : First 2 cases of use in humans for single-gene disorder.
FMO3 Gene Therapy
It's unknown when FMO3 would be trialed by a health system or private company.
All the 'obvious serious' genetic disorders are ;likely to be at the front of the queue.
They still need to see how the 2 cases get on (but looking good after around 3 months).
They will need to find out delivery methods, and spend millions on research etc.
But an excellent start, and FMO3 should (in most cases) be straightforward base editing (not always).
Links
Link (New Atlas) : Encouraging early results from first human CRISPR gene therapy trials
NIH Directors Blog post
"As groundbreaking as CRISPR/Cas9 has been for editing specific genes, the system has its limitations. The initial version is best suited for making a double-stranded break in DNA, followed by error-prone repair. The outcome is generally to knock out the target. That’s great if eliminating the target is the desired goal. But what if the goal is to fix a mutation by editing it back to the normal sequence?
The new prime editing system, which was described recently by NIH-funded researchers in the journal Nature, is revolutionary because it offers much greater control for making a wide range of precisely targeted edits to the DNA code, which consists of the four “letters” (actually chemical bases) A, C, G, and T "
Link
CRISPR Therapeutics (done the 2 cases) : Press Release Link
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