Gene Therapy News Archive

Scientists overcome hurdles to design human artificial chromosomes

Singularity Hub - 26-Mar-2024

New technique delivers more genetic material, paving way for safer & effective gene therapies

Nanoscope's gene therapy offers hope for patients blinded by inherited retinal disease

FierceBiotech - 26-Mar-2024

Phase 2 trial results show significant vision improvement, plans for FDA approval

Gene therapy for rare brain disease sets a new pricing record at $4.25 million

FiercePharma - 20-Mar-2024

Its record-breaking price raises questions about affordability and access

Novel approach effectively silences cholesterol genes in mice for a year

News Medical - 01-Mar-2024

This could lead to long-lasting cholesterol treatments without genetic damages

Invisible stem cells treat disease without triggering immune response

Nature - 28-Feb-2024

Genetically-modified stem cells avoid immune rejection, paving way for universal therapies for everyone

Diabetic monkeys regain insulin independence after transplant of unique insulin cells

Live Forever Club - 23-Feb-2024

This could lead to a safer and better treatment for type 1 diabetes.

RNA editing therapies for genetic diseases signify medical research progress

Nature - 16-Feb-2024

Unlike editing DNA itself, this new approach makes temporary changes to RNA

Rejuvenate Bio partners with animal company to develop gene therapy for canine osteoarthritis

Biospace - 14-Feb-2024

Single dose injection targets root causes, offering long-term relief & improved quality of life

Fractyl raised $110 million to develop treatments for obesity and diabetes

Longevity Technology - 05-Feb-2024

They picked RJVA-001, a gene therapy, as their first clinical candidate

Dresden merges recombinases & DNA-binding domains for tailored gene editing

Medical Xpress - 31-Jan-2024

The research offers safer and more precise ways to treat genetic disorders

A CRISPR gene editing technique has cured hereditary angioedema in a single-dose

New Scientist - 31-Jan-2024

It demonstrated a remarkable cure rate, rather than merely offering a treatment

MeiraGTx at the forefront of gene therapy for cancer, obesity, and vision loss

Evening Standard - 21-Dec-2023

London biotech promises revolutionary therapies by replacing faulty genes & flipping gene switches

ARCUS gene editing technology effectively eliminates mutant mitochondrial DNA

Biospace - 04-Dec-2023

Potential therapeutic approach for mitochondrial and other genetic diseases

UK's approval of casgevy paves the way for gene-edited treatment of blood disorders

BBC - 16-Nov-2023

Casgevy offers hope for patients with sickle cell disease and beta thalassemia

Single-dose gene-editing therapy offers permanent solution to high cholesterol levels

New Scientist - 14-Nov-2023

Early results show promise in eliminating the need for lifelong statins

A new gene therapy treatment has helped deaf children hear for the first time

MIT Technology Review - 27-Oct-2023

Treatment delivers a working copy of a missing gene to the inner ear

Beyond CRISPR: Fanzors, a new era of programmable DNA editing tools

Massachusetts Institute of Technology (MIT) - 13-Oct-2023

Scientists discover a vast array of RNA-guided enzymes in diverse organisms

Broken string secures $15m to make cell and gene therapy safer, more accessible

Longevity Technology - 19-Sep-2023

Company's INDUCE-seq platform could revolutionise cell & gene therapy efficiency

Amber Bio secures $26M funding to advance new gene editing technologies

Longevity Technology - 07-Aug-2023

By editing RNA instead of DNA, the company aims to treat multiple undruggable genetic diseases safely

Voyager's new gene therapy targets amyloid plaques in Alzheimer's disease

FierceBiotech - 04-Aug-2023

New treatment approach could be more effective in lowering the risk of serious side effects

Novel method facilitates single-cell expansion of gene edited stem cells

Lifespan.io (LEAF) - 17-Jul-2023

Its polymer-supplemented culture system enables cell expansion without harmful mutations

Gene therapy restores partial red colour vision in total colour blindness patients

New Scientist - 10-Jul-2023

Despite the limited improvement, gene therapy offers hope for future breakthroughs in restoring vision

Game-changing bacteriophage could revolutionize gene therapy

New Scientist - 30-May-2023

Ability to carry vast amounts of DNA and other molecules promises substantial advancements in gene and cell therapies

Therapeutic applications of base editing clears girl's leukemia

BBC - 11-Dec-2022

Base editing can soon be a part in taking control of our genomes to fight genetic diseases

CRISPR based PASTE (Programmable Addition via Site-specific Targeting Elements) tool designed

MIT Media Lab - 24-Nov-2022

The new PASTE tool replaces faulty genes in a safer and more efficient way

Jeune Aesthetics moves to the Phase 2 trial for fine lines and wrinkles

Biospace - 17-Nov-2022

Targeted gene-delivery hold potential in replenishing the skins key protein, to treat fine lines

Breakthrough gene therapy offers cure for a rare bleeding disorder heamophilia

BBC - 21-Jul-2022

Trial results were positive but cautioned the use until proven safe and effective

HTR in Drosophila somatic cells using nikase and CRISPR genetic editing tools

Genetic Engineering & Biotechnology News (GEN) - 05-Jul-2022

CRISPR genetic editing tools may be helpful in genetic repair

Lifespan of middle-aged mice increased by a third with telomerase gene therapy

CISION PR Newswire - 19-May-2022

Treatment used proprietary CMV vector which is able to deliver multiple genes into the nucleus

Viral-vector gene therapies could unlock healthy life extension in humans

Lifespan.io (LEAF) - 12-May-2022

Lifespan of mice greatly extended even when treatment only started at the equivalent of 56 years old

Verve Therapeutics' gene editing therapy may cure heart attacks once and for all

Bloomberg - 06-May-2022

Trials underway to test the safety, but affordability and acceptance of gene therapy are major concerns

Gene therapy gel heals wounds in people with rare genetic skin condition

New Scientist - 28-Mar-2022

Positive results in clinical trials provide hope for conditions that currently have no specific treatments

Long-term partial reprogramming reversed ageing process in mice

The Guardian - 07-Mar-2022

Yamanaka factors restored youthfullness in aged cells, raised hope for new anti-ageing therapies

NHS offers the world's most expensive drug for children with a rare disease

BBC - 04-Feb-2022

Libmeldy is life-saving gene therapy now available for use in England

Gene-editing technology, CRISPR, selectively kills drug-resistant superbugs

Deutsche Welle (DW) - 10-Nov-2021

A single dose of CRISPR Cas system cleared 99.9% of harmful bacterial in mice

Turning off abnormal genes may cure crippling genetic disease

BBC - 21-Oct-2021

Potential gene silencing approach in porphyria and possibly for many other diseases in the future

FDA puts clinical hold on Allogenic CAR T therapy due to safety concerns

FierceBiotech - 08-Oct-2021

Chromosome abnormality reported in a clinical trial raises doubts on patient safety

OMEGA, a new powerful gene-editing system similar to CRISPR or so much better

New Atlas - 16-Sep-2021

Potential RNA-guiding enzymes that are capable of editing and modifying DNA in human cells

Centaura's human artificial chromosome to treat complex diseases, including ageing

Medium - 10-Sep-2021

A breakthrough technology focusing on effective gene therapies for healthy longevity

Lifespan.io interviews George Church about gene therapy and longevity

Lifespan.io (LEAF) - 28-Jul-2021

Discusses animal trials, potential human trials, reeducating the FDA and improvements in technology

New CRISPR gene-editing drug for a rare protein-misfolding disorder

pharmaphorum - 28-Jun-2021

Phase 1 trial reports, single dose of the drug cuts protein levels by 87% in ATTR amyloidosis

Brain can learn to see again using algae proteins

BBC - 24-May-2021

Optogenetics bring back vision partially with light sensing proteins - new hope for the blind

New gene therapy technique, CopyCatcher, more accurate in fruit flies

SciTechDaily - 11-May-2021

Targets precise location of mutated gene rather than tagging on anywhere

Gene therapy preserved memory and learning in mice with Alzheimer's

Lifespan.io (LEAF) - 11-May-2021

Boosting a vital protein, Caveolin-1, this new technique offers hope against neurodegenerative diseases

Inherited blindness can now be cured with gene therapy

BBC - 29-Apr-2021

A breakthrough gene therapy restores eyesight in patients with rare eye disease

Latest CRISPR editing method can turn on/off gene expression

Phys.org - 09-Apr-2021

Allows to silence bad genes without modifying the DNA sequence and it is fully reversible

Gene therapy for Alzheimer's disease enters human clinical trial

Forbes - 12-Mar-2021

Virus carries gene encoding protein into the brain -possible breakthrough treatment for neurodegenerative diseases

Single gene therapy injection halts spinal muscular atrophy

BBC - 08-Mar-2021

Becomes most expensive drug ever approved for the NHS - but no lifetime costs

CRISPR cuts cholesterol by 57% with just one shot

Phys.org - 01-Mar-2021

Tiny lipid particles with gene editing machinery selectively targets liver and reduces cholesterol in mice

A look at the government consultation on genetic technologies in agriculture

Nuffield Council on Bioethics - 04-Feb-2021

Two kinds of question that people ask about developments in life sciences and biotechnology:

Deactivating ageing gene KAT7 to slow down ageing

Reuters - 20-Jan-2021

A potential therapeutic target for ageing, but still a long way to human trials

A gene editing tool used to treat progeria in mice

Science Magazine - 06-Jan-2021

Hope to launch a clinical trial in kids soon using this approach

Gene therapy prevented deafness in mice with genetic defects

ISRAEL21c - 04-Jan-2021

Scientists to use this approach & treat deafness due to other mutations also

CRISPR's gene editing therapy finds cure for blood cancer

STAT - 05-Dec-2020

Early data from Vertex & CRISPR showed promising results to cure blood disorders

MIT professor Richard Young explains how Omega regulates gene expression

Cell and gene - 01-Dec-2020

Using Epigenomic Controllers to alter DNA transcription rather than the genes themselves

A novel gene editing tool to fight aggressive cancers

Medical Xpress - 18-Nov-2020

Uses CRISPR technology to target and destroy cancer cells

A potential therapy for blinding retinal diseases

National Institutes of Health (NIH) - 03-Nov-2020

Researchers engineer novel protein that restores vision in blind mice via gene therapy

Tiny lipid bubbles could deliver gene editing kit to your tissues

Phys.org - 08-Oct-2020

Will make it easier to target treatment to specific locations in the body

Old mice show improved muscle function with gene therapy

Lifespan.io (LEAF) - 27-Aug-2020

A single injection could treat motor diseases of the aged but needs more human studies

90 % of herpes simplex virus 1 eliminated in infected cells

Lifespan.io (LEAF) - 19-Aug-2020

Successful in mice, new approach may ease immune burden of herpes in humans in the future

$95M raised by Taysha for clinical trials of four new gene therapies

FierceBiotech - 05-Aug-2020

Company has gathered support from investors to quickly lead therapies to clinic then market

Scientists go where no gene-editing tool has gone before

Lifespan.io (LEAF) - 14-Jul-2020

DddA able to target mitochondrial DNA (mtDNA) by splitting in half and hitching a ride

A gene editing tool to treat mitochondrial mutations

The Scientist - 09-Jul-2020

Derived from bacterial toxin - can make single base pair changes

Company introduces 'gene writing', to surpass CRISPR's limitations

Wired - 07-Jul-2020

New class of gene editors set to remove hurdles of traditional gene therapy

Gene editing lowered blood level cholesterol and triglycerides in monkeys

New York Times - 27-Jun-2020

Could prevent not only the complications but the heart disease per se in the future

CRISPR gene editing to cure beta thalassaemia and sickle cell disease

New Scientist - 12-Jun-2020

Didn't change the inherited faulty gene, but gene to switch on fetal haemoglobin production

Phase 1 clinical trial for type 1 diabetes cell therapy comes closer

Medical Xpress - 03-Jun-2020

A world-first novel technology to treat the disease is now within reach

Automated manufacturing of custom-engineered cell lines

News Medical - 20-May-2020

This technology is very promising and has an enormous potential

Follistatin gene therapy to combat osteoarthritis and obesity

Next Big Future - 16-May-2020

This new approach doubled muscle mass in mice, reduced its fat

Mapping gene expression regulation in yeast to understand ageing

Google AI Blog - 29-Apr-2020

Made possible by Calico's research expertise and Google AI's machine learning tech

CRISPR-Cas9 variants, key to overcoming major limitation in gene editing system

Lifespan.io (LEAF) - 31-Mar-2020

Entire genome now targetable, open to interventions for use in combating diseases

A global commission laid out roadmap for gene-editing trials on human embryo

Wired - 09-Mar-2020

Strict ethical guidelines to avoid undesirable outcomes in gene-edited babies

Fixing the genes to cure inherited blindness

Guardian - 04-Mar-2020

Crispr gene editing is a great but a dangerous tool

First gene therapy for a rare eye disease, now NHS-approved

BBC - 17-Feb-2020

This life-changing new gene therapy will halt sight loss and improve vision

CRISPR gene editing to help immune cells target and kill cancer

New Scientist - 06-Feb-2020

First CRISPR cancer trial in the world publish its promising findings

Chinese scientist tried genome editing on embryos

Guardian - 31-Dec-2019

Research of He Jiankui was a violation of medical regulations

Scientists identify multiple genes responsible for keeping cancer cells alive

Independent - 20-Dec-2019

If combined they can form the largest database of genetic information on cancer

NIH and Gates Foundation to invest $100 million each to develop gene therapies

Time Magazine - 24-Nov-2019

New hope for the 37.9 million people worldwide living with HIV/AIDS

CRISPR technology edited plant cells after being sprayed on their leaves

New Scientist - 01-Nov-2019

Can get into animal cells, so I wonder if this could be used with humans in future?

Prime editing as a way of "editing" genes

BBC - 21-Oct-2019

There are around 75,000 mutations caused by DNA errors and prime editing can correct 89% of them

Direct cell to cell transformation without a stem cell stage

TechCrunch - 14-Oct-2019

Analyses a huge amount of data without the help of an artificial intelligence

Drug invented for a girl with Batten disease

BBC - 12-Oct-2019

Doctors from Boston sequenced in detail whole genome of Mila and found the disease-causing mutation

New gene therapy to treat AMD will last a lifetime

ScienceDaily - 11-Oct-2019

A better option than having injections in to the eye once a month

Cervical cancer successfully cured with gene editing in mice

Medical Xpress - 10-Oct-2019

If the right genes to tackle are identified, this could be the cure for all cancers

Nonviral gene therapy: the safer option and full of potential

Phys.org - 24-Sep-2019

Could it be used to increase the crop yield to fight against world hunger?

New treatment for inherited retinal dystrophies

BBC - 04-Sep-2019

Gene therapy presents injecting the healthy gene directly in the eye to produce working protein

Treatment with immune system caused remission in incurable leukaemia

Independent - 02-Sep-2019

CAR-T therapy gives hope for the children for whom the standard therapies don't work

Pirating a $7000 version of a million-dollar gene therapy

MIT Technology Review - 30-Aug-2019

This could be beneficial if things go well, but dangerous otherwise

Salk scientists developed a new genome-editing tool tested on mice

Salk Institute - 23-Aug-2019

New powerful tool is developed for targeting the non-coding areas of DNA

Human trials using CRISPR to cure genetically inherited blindness

Futurism - 30-Jul-2019

There are at least 13 types of LCA - this trial is aimed at treating LCA 10

First attempt to use CRISPR tool to treat a genetic disorder

NPR - 29-Jul-2019

Cells are being taken from patients' bone marrow and genetically modified with CRISPR

NHS gives the green-light to a “gene-silencing” drug for the first time

BBC - 09-Jul-2019

It took thirteen years for RNA interference technology to come to this stage

Crispr injection could reduce chance of heart attack

Guardian - 10-May-2019

Verve Therapeutics treatment targets PCSK9 gene

Interfering with enzyme production reduces crippling pain

BBC - 13-Apr-2019

Gene silencing rather than gene therapy

Gene therapy makes non-vision cells light sensitive

University of California, Berkeley - 15-Mar-2019

This would be a stunning result if repeated in humans

Human tissue regeneration possible with complete axolotl genome map

Gizmodo - 24-Jan-2019

Understanding regeneration in axolotls will be a paradigm shift for medicine

New gene therapies having big impact

Guardian - 19-Jan-2019

Successful treatment of retinitis pigmentosa and spinal muscular atrophy

What Happens When CRISPR Backfires?

Seeker - 25-Sep-2018

Recent research revealed novel problems with the CRISPR technique, delaying human trials

Drug approved as alternative method to CRISPR

Singularity Hub - 21-Aug-2018

Patisiran is a new drug capable of silencing genes based on RNA interference technology

Caution Regarding CRISPR Gene Therapies

Singularity Hub - 23-Jul-2018

Newly found lack of precision of CRISPR delays plans for human trials

Gene therapy reverses rat's paralysis

BBC - 14-Jun-2018

Study used virus to deliver genetic information that reduced scar tissue in the spinal cord

Gene Therapies to Greatly Reduce the Incidence of Cardiovascular Disease

Fight Aging! - 09-Mar-2018

Gene therapy could disable genes that sustain levels of lipids in the bloodstream.

CRISPR Toolkit Expands With Trio of New Tricks

Singularity Hub - 27-Feb-2018

New CRISPR technologies are CAMERA, DETECTR, and SHERLOCK.

Haemophilia A gene therapy results 'mind-blowing'

BBC - 14-Dec-2017

Haemophilia prevents production of a protein needed to stop bleeding.

Gene Therapies as a Basis to Control Aging

Fight Aging! - 04-Dec-2017

In principle, a gene therapy can do everything a drug can.

FDA Target Rogue Gene Therapy Clinics

New York Times - 16-Nov-2017

FDA issued new guidelines for expedited review of stem cell and gene therapies.

First ever attempt at gene editing inside the body

Guardian - 15-Nov-2017

Patient lacks a gene that makes an enzyme that breaks down certain carbohydrates.

Genetically modified skin in life-saving treatment

BBC - 08-Nov-2017

Junctional epidermolysis bullosa DNA lacks the instructions to stick epidermis to the dermis.

17 new genes linked to longer lifespan identified

Times Now News - 08-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.

Scientists Discover a Key to a Longer Life in Male DNA

New York Times - 16-Jun-2017

Growth hormone receptors is linked to an increase in life span of about 10 years among men.

Ageing is a Disease. Gene Therapy Could be the Cure

WIRED - 20-Apr-2017

Most age related diseases are a symptom of cellular malfunction.

Gene therapy cures boy of sickle cell disease

New Scientist - 01-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.

Qatar leading ‘ambitious’ genome project in Gulf

Gulf Times - 06-Jan-2017

Qatar Genome Programme (QGP) focuses on mapping the population as well as treating common and rare diseases.

Possible way to stop activation of proteins known to cause age-related diseases

News Medical - 15-Dec-2016

Scientists have discovered that it is possible to stop the activation of a group of proteins – NADPH Oxidase.

CRISPR Gene Editing Cures Mice of Sickle Cell Disease

Futurism - 12-Oct-2016

CRISPR used to correct mutated hematopoietic stem cells.

Target Genes for Slowing Aging

Fight Aging! - 06-Jun-2016

75 potential targets for gene therapy in the near future.

Could mRNA Overtake all other Biologicals in Medicine?

Labiotech - 05-Jan-2016

What if we could send the body an instruction to produce its own drug?

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.

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.

Gene therapy restores hearing in deaf mice

Kurzweil Network - 13-Jul-2015

Targeted delivery of functioning TMC1 gene to hair cells in cochlea using engineered virus AAV1.