Showing 13 to 24 of 43 results
Gene Therapy Shows Promise for Long-Term Osteoarthritis Pain Relief
A Phase 1 clinical trial at the Mayo Clinic successfully tested a gene therapy for osteoarthritis in nine patients, showing reduced pain and improved knee mobility for at least a year after a single injection of a genetically-modified virus delivering an anti-inflammatory molecule, with only minor s...
Gene Therapy Shows Promise for Long-Term Osteoarthritis Pain Relief
A Phase 1 clinical trial at the Mayo Clinic successfully tested a gene therapy for osteoarthritis in nine patients, showing reduced pain and improved knee mobility for at least a year after a single injection of a genetically-modified virus delivering an anti-inflammatory molecule, with only minor s...
Progress
52% Bias Score
\£10 Million Grant Funds Project to Create Synthetic Human Chromosome
The Synthetic Human Genome Project (SynHG) received \£10 million to create a synthetic human chromosome within 5-10 years, aiming to advance genome synthesis technology for disease treatment and research, while raising ethical concerns about potential misuse.
\£10 Million Grant Funds Project to Create Synthetic Human Chromosome
The Synthetic Human Genome Project (SynHG) received \£10 million to create a synthetic human chromosome within 5-10 years, aiming to advance genome synthesis technology for disease treatment and research, while raising ethical concerns about potential misuse.
Progress
16% Bias Score
Gene Therapy Shows Promise in Frontotemporal Dementia Trials
A British company, AviadoBio, is conducting clinical trials of a gene therapy (AVB-101) for frontotemporal dementia (FTD-GRN), a genetic form of dementia, infusing a functional gene copy directly into the brain to restore progranulin protein levels; early trial data is expected next year.
Gene Therapy Shows Promise in Frontotemporal Dementia Trials
A British company, AviadoBio, is conducting clinical trials of a gene therapy (AVB-101) for frontotemporal dementia (FTD-GRN), a genetic form of dementia, infusing a functional gene copy directly into the brain to restore progranulin protein levels; early trial data is expected next year.
Progress
44% Bias Score
Retinoic Acid's Role in Axolotl Limb Regeneration: Implications for Human Healing
Researchers discovered that retinoic acid directs limb regeneration in axolotls, controlling which cells regenerate and how; this finding could lead to new human healing methods and gene therapies, as published in Nature Communications.
Retinoic Acid's Role in Axolotl Limb Regeneration: Implications for Human Healing
Researchers discovered that retinoic acid directs limb regeneration in axolotls, controlling which cells regenerate and how; this finding could lead to new human healing methods and gene therapies, as published in Nature Communications.
Progress
24% Bias Score
Gene Editing Successfully Treats Baby with Rare Genetic Disorder
US doctors successfully used a customized gene-editing therapy to treat a baby, KJ, with severe CPS1 deficiency, a rare genetic disorder, showing early signs of success and highlighting the potential of gene editing for treating various genetic diseases.
Gene Editing Successfully Treats Baby with Rare Genetic Disorder
US doctors successfully used a customized gene-editing therapy to treat a baby, KJ, with severe CPS1 deficiency, a rare genetic disorder, showing early signs of success and highlighting the potential of gene editing for treating various genetic diseases.
Progress
40% Bias Score
Gene Editing Therapy Shows Promise in Treating Rare Genetic Disorder
Nine-month-old KJ Muldoon, diagnosed with severe CPS1 deficiency, a rare genetic disorder affecting one in a million babies, is thriving after receiving a custom CRISPR-based gene editing therapy developed in six months by researchers at Children's Hospital of Philadelphia and Penn Medicine.
Gene Editing Therapy Shows Promise in Treating Rare Genetic Disorder
Nine-month-old KJ Muldoon, diagnosed with severe CPS1 deficiency, a rare genetic disorder affecting one in a million babies, is thriving after receiving a custom CRISPR-based gene editing therapy developed in six months by researchers at Children's Hospital of Philadelphia and Penn Medicine.
Progress
40% Bias Score
Gene Therapy Restores Sight in Usher Syndrome Patient
A 38-year-old Italian man with Usher syndrome type 1b regained his sight after an experimental gene therapy at the University of Campania in July 2024; seven more patients underwent the procedure, showing promising results and paving the way for broader application.
Gene Therapy Restores Sight in Usher Syndrome Patient
A 38-year-old Italian man with Usher syndrome type 1b regained his sight after an experimental gene therapy at the University of Campania in July 2024; seven more patients underwent the procedure, showing promising results and paving the way for broader application.
Progress
20% Bias Score
Tetraneuron's Novel Gene Therapy Targets Alzheimer's Disease by Restoring Neuronal Function
Tetraneuron, based on research from the Cajal Institute in Madrid, is developing a gene therapy for moderate-to-severe Alzheimer's disease by targeting the transcription factor E2F4, showing promising results in animal models and aiming for human trials in 2026.
Tetraneuron's Novel Gene Therapy Targets Alzheimer's Disease by Restoring Neuronal Function
Tetraneuron, based on research from the Cajal Institute in Madrid, is developing a gene therapy for moderate-to-severe Alzheimer's disease by targeting the transcription factor E2F4, showing promising results in animal models and aiming for human trials in 2026.
Progress
32% Bias Score
Retinoic Acid Directs Limb Regeneration in Axolotls, Offering Clues for Human Healing
Researchers discovered that retinoic acid, a molecule also present in retinol, controls limb regeneration in axolotls by signaling injured cells; controlling its levels through the enzyme CYP26B1 is key for precise regrowth, potentially leading to new human healing methods.
Retinoic Acid Directs Limb Regeneration in Axolotls, Offering Clues for Human Healing
Researchers discovered that retinoic acid, a molecule also present in retinol, controls limb regeneration in axolotls by signaling injured cells; controlling its levels through the enzyme CYP26B1 is key for precise regrowth, potentially leading to new human healing methods.
Progress
20% Bias Score
In-Utero Gene Therapy Shows Promise in Preventing Inherited Diseases
New research demonstrates successful in-utero gene therapy in animal models preventing spinal muscular atrophy, with early human trials showing promise and raising ethical questions about germline modification.
In-Utero Gene Therapy Shows Promise in Preventing Inherited Diseases
New research demonstrates successful in-utero gene therapy in animal models preventing spinal muscular atrophy, with early human trials showing promise and raising ethical questions about germline modification.
Progress
52% Bias Score
Personalized Gene Therapy Shows Promise in Treating Baby with Rare Metabolic Disorder
In February 2025, a baby born with a life-threatening metabolic disorder received a personalized gene therapy in the US, showing significant health improvements and enabling the baby to return home, representing a potential breakthrough for treating rare genetic diseases with personalized base editi...
Personalized Gene Therapy Shows Promise in Treating Baby with Rare Metabolic Disorder
In February 2025, a baby born with a life-threatening metabolic disorder received a personalized gene therapy in the US, showing significant health improvements and enabling the baby to return home, representing a potential breakthrough for treating rare genetic diseases with personalized base editi...
Progress
40% Bias Score
Losartan Shows Promise for Butterfly Skin, but Funding for Clinical Trial Remains a Challenge
Losartan, a hypertension drug, shows promise in treating epidermolysis bullosa (butterfly skin) lesions, but a €5-10 million clinical trial is needed for EMA approval, highlighting challenges in repurposing drugs for rare diseases.
Losartan Shows Promise for Butterfly Skin, but Funding for Clinical Trial Remains a Challenge
Losartan, a hypertension drug, shows promise in treating epidermolysis bullosa (butterfly skin) lesions, but a €5-10 million clinical trial is needed for EMA approval, highlighting challenges in repurposing drugs for rare diseases.
Progress
36% Bias Score
Showing 13 to 24 of 43 results