Gene Therapy

Restoring the body with gene therapy.

Latest News

Caution Regarding CRISPR Gene Therapies – Singularity Hub – 23-Jul-2018
CRISPR can cause more unintended damage than thought.
Enzyme cuts a cell’s DNA and relies on the cell to reconnect.
Can cause large deletions or rearrangements of DNA around the target site.
Fears this kind of damage to DNA could lead to cancer.
Has gone unnoticed as looking for small mutations.
Changes in the DNA have been seriously underestimated

Gene therapy reverses rat’s paralysis – BBC News – 14-Jun-2018
King’s College London repaired damage in spinal cord of rats.
Body repairs a wounded spinal cord with scar tissue.
But scar acts like a barrier to new nerve connections.
Used a virus to alter enzyme called chondroitinase.
Animals regained use of their front paws after the gene therapy.

Gene Therapies to Greatly Reduce the Incidence of Cardiovascular Disease – Fight Aging – 9-Mar-2018
Gene therapy could disable genes that sustain levels of lipids in the bloodstream.
Number of credible targets including PCSK9, ANGPTL3, ANGPTL4, and ASGR1.
Reducing lipid levels in the bloodstream slows the development of CVD.
Treatments will initially have a low effectiveness in terms of proportion of cells transfected.

CRISPR Toolkit Expands With Trio of New Tricks – Singularity Hub – 27-Feb-2018
New CRISPR technologies are CAMERA, DETECTR, and SHERLOCK.
CAMERA reads out for the cell’s historical exposure to antibiotics or toxins.
SHERLOCK uses sacrificial RNA molecules detect presence of virus or cancer DNA.
DETECTR could turn CRISPR into a sensitive one-step diagnostic tool.
More research is exploring CRISPR applications beyond gene therapy.
“The killer app is still to come” – Dr. Dave Savage.

Haemophilia A gene therapy results ‘mind-blowing’ – BBC News – 14-Dec-2017
Haemophilia prevents production of a protein needed to stop bleeding.
Even the impact of walking can lead to bleeding in joints.
Sufferers typically need injections of factor VIII every other day for life.
Trial resulted in 11 of 13 participants producing near-normal levels of the protein.
Gene therapies are very expensive but factor VIII injections cost £100,000 per year.
Haemophilia effects 2,000 people in the UK.
COMMENT: cost of gene therapies will reduce as they become more common

Gene Therapies as a Basis to Control Aging – Fight Aging – 4-Dec-2017
In principle, a gene therapy can do everything a drug can.
Should perform more effectively and more accurately.
The laziness of evolution has resulted in your program to not avoid dying.
Genes that work for longevity in smaller organisms can be reconfigured in humans.
Expect first dog trials in the next year or two.

17 new genes linked to longer lifespan identified – Times Now News – 8-Nov-2017
A team of researchers have discovered 17 new genes linked to longer lifespan, which they say could one day be targeted to help prolong human life.
The team studied 389,166 volunteers who took part in the UK Biobank, with confirmation in the US Health and Retirement Study and the Wisconsin Longitudinal Study.
The study found evidence to suggest that the genetic variants for average lifespan also influence exceptionally long life expectancy.

FDA Target Rogue Gene Therapy Clinics – NY Times – 16-Nov-2017
FDA issued new guidelines for expedited review of stem cell and gene therapies.
Will crack down on clinics offering unproven versions of those treatments.
Only two products that qualify as gene therapy have been approved.
Patients have been blinded after fat-derived cells were injected into their eyes.
Previoulsy practitioners argued that they do not come under the agency’s jurisdiction.

First ever attempt at gene editing inside the body – The Guardian – 15-Nov-2017
Patient lacks a gene that makes an enzyme that breaks down certain carbohydrates.
Symptoms include distorted facial features, hearing loss, breathing trouble and cognitive problems.
Zinc finger nucleases work like molecular scissors that replace a specific piece of DNA.
Only 1% of liver cells would have to be corrected to successfully treat the disease.
It becomes part of your DNA and is there for the rest of your life.
There is no way to erase any mistakes the editing might cause.
Tests will confirm whether it is working in three months.

Genetically modified skin in life-saving treatment – BBC News – 08-Nov-2017
Junctional epidermolysis bullosa DNA lacks the instructions to stick epidermis to the dermis.
Genetic disease leaves skin as fragile as a butterfly’s wings.
4 sq cm patch of skin was taken and its DNA repaired in the laboratory.
Genetically modified skin cells were grown to make skin grafts totalling 0.85 sq m.
After 21 months skin is functioning normally with no sign of blistering.

Scientists Discover a Key to a Longer Life in Male DNA – NY times – 16-Jun-2017
Growth hormone receptors is linked to an increase in life span of about 10 years among men.
This common genetic mutation did not seem to have any effect on women.
Mutation was present in 12 percent of the men over age 100 – 3 times higher than in 70-year-olds.
Present in roughly the same fraction for both age groups in women.

Ageing is a Disease. Gene Therapy Could be the Cure – WIRED – 20-Apr-17
Most age related diseases are a symptom of cellular malfunction.
Gene therapy allows us to replace or block faulty genes, and up-regulate beneficial ones.
Some strains of mice have had life extended 5x using gene modulations.
Liz Parrish, CEO of company, took 2 leading therapies.
1. telomerase gene therapy to lengthen telomeres
2. myostatin inhibitor to prevent age-associated muscle loss
Saw positive results in blood biomarkers
Starting safety trials this year.

Gene therapy cures boy of sickle cell disease – New Scientist – 1-Mar-2017
People with sickle cell disease make abnormal versions of haemoglobin – distorting red blood cells into a sickle-shape that can get stuck in blood vessels.
Bone marrow stem cells removed, treated, and replaced in boy’s body.
Medication and symptom free two years later.
Team has treated seven other patients, who are showing “promising” progress.
Treatment could work for everyone with the disorder.

Qatar leading ‘ambitious’ genome project in Gulf – Gulf Times – 6-Jan-2017
Qatar Genome Programme (QGP) focuses on mapping the population as well as treating common and rare diseases.
Pilot phase has seen sequencing of 3,000 genomes of Qatari nationals.
Sequencing genomes of 10% of the population is good enough to generate enough data that could be applied on the entire population of a nation.
In collaboration with Pathway towards Personalised Medicine (PPM) to encourage local research on genomics.

Possible way to stop activation of proteins known to cause age-related diseases – – 15-Dec-2016
Scientists have discovered that it is possible to stop the activation of a group of proteins – NADPH Oxidase.
NADPH Oxidase complex attacks blood vessels, the heart lining, joints and the brain when placed under metabolic stress, causing most of the diseases of ageing.
But also needed for cell communication.
Researchers have found a single nucleotide polymorphism (SNP) that protects against cardiovascular disease and also affects the activation of NADPH oxidase.
Drugs could be designed to prevent the activation process in conditions of stress, without affecting cell health.

CRISPR Gene Editing Cures Mice of Sickle Cell Disease – Futurism – 12-Oct-2016
CRISPR used to correct mutated hematopoietic stem cells.
Corrected blood stem cells produced healthy hemoglobin.
Engineered stem cells remained in circulation for at least four months.
Safety analyses needed before human trials.

Target Genes for Slowing Aging – Fight Aging! – 6-Jun-2016
75 potential targets for gene therapy in the near future.
Rapid advances in genetic editing technologies have made gene therapies cheaper and easier.
Focus is on compensatory therapies for aging.
List omits inherited disorders based on single faulty genes.

Could mRNA Overtake all other Biologicals in Medicine? – Labiotech – 5-Jan-2016
What if we could send the body an instruction to produce its own drug?
RNA strand seems easier to produce and characterize.
Better immune response as antigens are produced in the host cell.
Only targeted cells will express the protein.
Still major challenges to be overcome.
Three main players: Moderna, Urevac, BioNTEch
2 projects already in Phase II clinical trials.

Genes that help you live to 100 – New Scientist – 30-Dec-2015
Searching the genomes of centenarians these genes are most clearly associated with lifespan.
20 per cent of lifespan is genetic, possibly more for living to be extremely old.
Variations are common in the general population, but centenarians seem to have fewer “bad” variants.
Discovered genes have different functions:
1. determines blood group
2. regulates cellular life cycles
3. involved in how the immune system recognises the body’s own cells
4. has been shown to extend lifespan in fruit flies

Can we extend our lifespan, one gene at a time? – Genetic Literacy Project – 27-Oct-2015
There are definitely environmental factors that influence longevity as well as genetic ones.
Genome wide association studies (GWAS) allow the genome to be searched for small variations, called single nucleotide polymorphisms, that occur more frequently in people who live longer.
No new variants for longevity have been conclusively proven in humans.

BioViva Moving Ahead With Human Gene Therapy for Telomerase Activation – Fight Aging! – 2-Oct-2015
First company to treat a person with gene therapy to reverse biological aging.
Patient to be monitored every year for 8 years.
May help determine if average telomere length is a marker of the progress of aging or whether telomere lengthening extends life – as seen in mice.

Gene therapy restores hearing in deaf mice – Kurzweil AI – 13-Jul-2015
Targeted delivery of functioning TMC1 gene to hair cells in cochlea using engineered virus AAV1.
TMC1 accounts for 4 to 8 percent of genetic deafness. Clinical trials of gene therapy for humans expected within 5 to 10 years.

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