Precision genetics: unlocking the secrets of mutation
Harvard University - 13-Nov-2024A revolutionary tool targets specific genes, advancing our understanding of evolution and disease
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A revolutionary tool targets specific genes, advancing our understanding of evolution and disease
They aim to use AI-driven capsid design for delivery for complex treatments
Tests in mice and human cells show long-lasting gene correction
A new breakthrough in nanotechnology offers a non-invasive way to access the cell's genetic material
A promising one-time treatment approach for arrhythmogenic cardiomyopathy
This revolutionary treatment could eliminate the need for lifelong blood transfusions
Company is particularly focusing on less invasive treatments for dry AMD and XLRP
The therapy is initially available at eight centers across the UK
Potential for advancements in medicine, agriculture, and other fields
Boosting IRAK-M protein protects retina in the eyes and paves way for potential AMD treatment
Company aims to improve protein production in genetic medicine
Therapy offers hope for a potential cure for certain types of hearing loss
Treatment shows improved blood flow, exercise tolerance and reduced symptoms
AI-powered platform predicts drugs for age-related diseases with 80% accuracy
This could lead to safer and more effective treatments for genetic diseases
New technique delivers more genetic material, paving way for safer & effective gene therapies
Phase 2 trial results show significant vision improvement, plans for FDA approval
This experimental therapy targets a common metabolic issue in Retinitis Pigmentosa
Its record-breaking price raises questions about affordability and access
This could lead to long-lasting cholesterol treatments without genetic damages
Genetically-modified stem cells avoid immune rejection, paving way for universal therapies for everyone
This could lead to a safer and better treatment for type 1 diabetes.
Unlike editing DNA itself, this new approach makes temporary changes to RNA
Single dose injection targets root causes, offering long-term relief & improved quality of life
They picked RJVA-001, a gene therapy, as their first clinical candidate
The research offers safer and more precise ways to treat genetic disorders
It demonstrated a remarkable cure rate, rather than merely offering a treatment
Patients’ vision and retinal health remained stable after six months
London biotech promises revolutionary therapies by replacing faulty genes & flipping gene switches
Tome Biosciences secures $213m to advance next-gen gene editing
Potential therapeutic approach for mitochondrial and other genetic diseases
Casgevy offers hope for patients with sickle cell disease and beta thalassemia
Early results show promise in eliminating the need for lifelong statins
Treatment delivers a working copy of a missing gene to the inner ear
Scientists discover a vast array of RNA-guided enzymes in diverse organisms
Company's INDUCE-seq platform could revolutionise cell & gene therapy efficiency
By editing RNA instead of DNA, the company aims to treat multiple undruggable genetic diseases safely
New treatment approach could be more effective in lowering the risk of serious side effects
Its polymer-supplemented culture system enables cell expansion without harmful mutations
Despite the limited improvement, gene therapy offers hope for future breakthroughs in restoring vision
Ability to carry vast amounts of DNA and other molecules promises substantial advancements in gene and cell therapies
Base editing can soon be a part in taking control of our genomes to fight genetic diseases
The new PASTE tool replaces faulty genes in a safer and more efficient way
Targeted gene-delivery hold potential in replenishing the skins key protein, to treat fine lines
Trial results were positive but cautioned the use until proven safe and effective
CRISPR genetic editing tools may be helpful in genetic repair
Treatment used proprietary CMV vector which is able to deliver multiple genes into the nucleus
Lifespan of mice greatly extended even when treatment only started at the equivalent of 56 years old
Trials underway to test the safety, but affordability and acceptance of gene therapy are major concerns
Positive results in clinical trials provide hope for conditions that currently have no specific treatments
Yamanaka factors restored youthfullness in aged cells, raised hope for new anti-ageing therapies
Libmeldy is life-saving gene therapy now available for use in England
A single dose of CRISPR Cas system cleared 99.9% of harmful bacterial in mice
Potential gene silencing approach in porphyria and possibly for many other diseases in the future
Chromosome abnormality reported in a clinical trial raises doubts on patient safety
Potential RNA-guiding enzymes that are capable of editing and modifying DNA in human cells
A breakthrough technology focusing on effective gene therapies for healthy longevity
Discusses animal trials, potential human trials, reeducating the FDA and improvements in technology
Phase 1 trial reports, single dose of the drug cuts protein levels by 87% in ATTR amyloidosis
Optogenetics bring back vision partially with light sensing proteins - new hope for the blind
Targets precise location of mutated gene rather than tagging on anywhere
Boosting a vital protein, Caveolin-1, this new technique offers hope against neurodegenerative diseases
A breakthrough gene therapy restores eyesight in patients with rare eye disease
Allows to silence bad genes without modifying the DNA sequence and it is fully reversible
Virus carries gene encoding protein into the brain -possible breakthrough treatment for neurodegenerative diseases
Becomes most expensive drug ever approved for the NHS - but no lifetime costs
Tiny lipid particles with gene editing machinery selectively targets liver and reduces cholesterol in mice
Two kinds of question that people ask about developments in life sciences and biotechnology:
A potential therapeutic target for ageing, but still a long way to human trials
Hope to launch a clinical trial in kids soon using this approach
Scientists to use this approach & treat deafness due to other mutations also
Early data from Vertex & CRISPR showed promising results to cure blood disorders
Using Epigenomic Controllers to alter DNA transcription rather than the genes themselves
Uses CRISPR technology to target and destroy cancer cells
Researchers engineer novel protein that restores vision in blind mice via gene therapy
Will make it easier to target treatment to specific locations in the body
A single injection could treat motor diseases of the aged but needs more human studies
Successful in mice, new approach may ease immune burden of herpes in humans in the future
Company has gathered support from investors to quickly lead therapies to clinic then market
DddA able to target mitochondrial DNA (mtDNA) by splitting in half and hitching a ride
Derived from bacterial toxin - can make single base pair changes
New class of gene editors set to remove hurdles of traditional gene therapy
Could prevent not only the complications but the heart disease per se in the future
Didn't change the inherited faulty gene, but gene to switch on fetal haemoglobin production
A world-first novel technology to treat the disease is now within reach
This technology is very promising and has an enormous potential
This new approach doubled muscle mass in mice, reduced its fat
Made possible by Calico's research expertise and Google AI's machine learning tech
Entire genome now targetable, open to interventions for use in combating diseases
Strict ethical guidelines to avoid undesirable outcomes in gene-edited babies
Crispr gene editing is a great but a dangerous tool
This life-changing new gene therapy will halt sight loss and improve vision
First CRISPR cancer trial in the world publish its promising findings
Research of He Jiankui was a violation of medical regulations
If combined they can form the largest database of genetic information on cancer
New hope for the 37.9 million people worldwide living with HIV/AIDS
Can get into animal cells, so I wonder if this could be used with humans in future?
There are around 75,000 mutations caused by DNA errors and prime editing can correct 89% of them
Analyses a huge amount of data without the help of an artificial intelligence
Doctors from Boston sequenced in detail whole genome of Mila and found the disease-causing mutation
A better option than having injections in to the eye once a month
If the right genes to tackle are identified, this could be the cure for all cancers
Could it be used to increase the crop yield to fight against world hunger?
Gene therapy presents injecting the healthy gene directly in the eye to produce working protein
CAR-T therapy gives hope for the children for whom the standard therapies don't work
This could be beneficial if things go well, but dangerous otherwise
New powerful tool is developed for targeting the non-coding areas of DNA
There are at least 13 types of LCA - this trial is aimed at treating LCA 10
Cells are being taken from patients' bone marrow and genetically modified with CRISPR
It took thirteen years for RNA interference technology to come to this stage
Verve Therapeutics treatment targets PCSK9 gene
Gene silencing rather than gene therapy
This would be a stunning result if repeated in humans
Understanding regeneration in axolotls will be a paradigm shift for medicine
Successful treatment of retinitis pigmentosa and spinal muscular atrophy
Recent research revealed novel problems with the CRISPR technique, delaying human trials
Patisiran is a new drug capable of silencing genes based on RNA interference technology
Newly found lack of precision of CRISPR delays plans for human trials
Study used virus to deliver genetic information that reduced scar tissue in the spinal cord
Gene therapy could disable genes that sustain levels of lipids in the bloodstream.
New CRISPR technologies are CAMERA, DETECTR, and SHERLOCK.
Haemophilia prevents production of a protein needed to stop bleeding.
In principle, a gene therapy can do everything a drug can.
FDA issued new guidelines for expedited review of stem cell and gene therapies.
Patient lacks a gene that makes an enzyme that breaks down certain carbohydrates.
Junctional epidermolysis bullosa DNA lacks the instructions to stick epidermis to the dermis.
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.
Growth hormone receptors is linked to an increase in life span of about 10 years among men.
Most age related diseases are a symptom of cellular malfunction.
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.
Qatar Genome Programme (QGP) focuses on mapping the population as well as treating common and rare diseases.
Scientists have discovered that it is possible to stop the activation of a group of proteins – NADPH Oxidase.
CRISPR used to correct mutated hematopoietic stem cells.
75 potential targets for gene therapy in the near future.
What if we could send the body an instruction to produce its own drug?
Searching the genomes of centenarians these genes are most clearly associated with lifespan.
There are definitely environmental factors that influence longevity as well as genetic ones.
Targeted delivery of functioning TMC1 gene to hair cells in cochlea using engineered virus AAV1.
First company to treat a person with gene therapy to reverse biological aging.