https://www.europesays.com/uk/70983/ Genetic medicine can leave people with rare mutations behind. But there’s new hope #121199049 #article #BirthDefects #Business #Cancer #ClinicalTrials #CysticFibrosis #GeneTherapy #Genetics #Health #Medication #philanthropy #Science #UK #UnitedKingdom
https://www.europesays.com/uk/52461/ Genetic medicine can leave people with rare mutations behind. But there’s new hope #APTopNews #BirthDefects #Business #Cancer #ClinicalTrials #CysticFibrosis #GarryCutting #GeneTherapy #Genetics #Health #Medication #PAStateWire #philanthropy #Science #UK #UnitedKingdom
A new blog by @pcublogue which adequately explains the trouble of not managing the diet well with PKU
www.phenylcetonurie.ca/p/phenylceto...
#pku #pcu #phenylketonuria #phenylcetonurie #health #healthcare #genetherapy
Phénylcétonurie : une maladie ...
https://www.europesays.com/uk/32997/ Report of a missense TJP2 variant associated to PFIC4 with a pronounced phenotypic variability: Focus on the structural effects on the protein level #ComputationalBiologyAndBioinformatics #GeneExpression #GeneFunction #GeneTherapy #Genetics #HumanGenetics #MolecularMedicine #NextGenerationSequencing #Paediatrics #Science #UK #UnitedKingdom
EUROPE
EU Plans New Life Sciences Strategy
EU to accelerate biotech & health innovation, aiming to catch up with US & China.
Focus on gene therapies, AI-powered personalized medicine & multinational clinical trials.
Plans to streamline drug regulations & boost funding for biotech startups.
#Biotech #AI #GeneTherapy #Healthcare #Europe
https://www.europesays.com/1890162/ Ocugen Provides Business Update with Fourth Quarter and #america #Biotechnology #business #Earnings #GeneTherapy #Nasdaq:OCGN #Ocugen #ophthalmology #UnitedStates #UnitedStatesOfAmerica #US #USA
PhD Student Position: Drug Development for Parkinson's Disease
Ben-Gurion University of the Negev
#PhDStudentPosition: #DrugDevelopment for #Parkinson'sDisease using #ProteinDesign, #ComputerAidedDrugDesign, #GeneTherapy, #iPSCs
See the full job description on jobRxiv: https://jobrxiv.org/job/ben-gurion-university-of-the-negev-27778-phd-student-posit...
https://jobrxiv.org/job/ben-gurion-university-of-the-negev-27778-phd-student-position-drug-development-for-parkinsons-disease/?feed_id=92530
Doctors in London have become the first in the world to cure blindness in children born with a rare genetic condition using a pioneering gene therapy.
The children had leber congenital amaurosis (LCA), a severe form of retinal dystrophy that causes vision loss due to a defect in the AIPL1 gene.
After treatment, four children can now see shapes, find toys, recognise their parents’ faces, and in some cases, even read and write.
The second revolution in gene therapy that has cured Javier, a child with a lethal error in the X chromosome.
An Italian foundation that helped a young boy overcome Wiskott-Aldrich syndrome becomes the first non-profit in the world to request approval of these innovative treatments from the European Medicines Agency and the American FDA.
I always love learning about new plasmid technologies (maybe I am biased from my Addgene days). Researchers from the University of Rochester developed a dual AAV vector system called StitchR that can deliver plasmids containing gene fragments that can be subsequently cut out and assembled into a functional gene. This helps overcome the small packaging capacity of AAVs.
Find out more from Drug Discovery News : https://buff.ly/4hwaxPb
PhD Student Position: Drug Development for Parkinson's Disease
Ben-Gurion University of the Negev
#PhDStudentPosition: #DrugDevelopment for #Parkinson'sDisease using #ProteinDesign, #ComputerAidedDrugDesign, #GeneTherapy, #iPSCs
See the full job description on jobRxiv: https://jobrxiv.org/job/ben-gurion-university-of-the-negev-27778-phd-student-posit...
https://jobrxiv.org/job/ben-gurion-university-of-the-negev-27778-phd-student-position-drug-development-for-parkinsons-disease/?feed_id=92033
Harnessing eukaryotic retroelement proteins for transgene insertion into human safe-harbor loci.
#GenomeEditing #GeneTherapy #TransposableElements
https://www.nature.com/articles/s41587-024-02137-y
PhD Student Position: Drug Development for Parkinson's Disease
Ben-Gurion University of the Negev
#PhDStudentPosition: #DrugDevelopment for #Parkinson'sDisease using #ProteinDesign, #ComputerAidedDrugDesign, #GeneTherapy, #iPSCs
See the full job description on jobRxiv: https://jobrxiv.org/job/ben-gurion-university-of-the-negev-27778-phd-student-posit...
https://jobrxiv.org/job/ben-gurion-university-of-the-negev-27778-phd-student-position-drug-development-for-parkinsons-disease/?feed_id=91788
Hope for those with #SickleCell disease! 
A potential functional cure for people in #England living with #SCD has been approved for use in the #NHS.
It is a form of one-time #GeneTherapy, & researchers are having a success rate of 96.6% in patients who received the treatment.
It is #hope for those (about 4000 people in the #UK) who are eligible for a stem cell transplant, but have no match. 
‘Groundbreaking’ sickle cell disease treatment approved for NHS use in England
Clinical trials find one-time gene therapy exa-cel offers ‘functional cure’ in 96.6% of patients
Trump ends program to lower prescription prices, make disease therapy available
https://www.alreporter.com/2025/01/21/trump-ends-program-to-lower-prescription-prices-make-disease-therapy-available/
#ExecutiveOrders
#Prescriptions
#Medicaid
#CellTherapy
#GeneTherapy
#Trump
$mgtx is currently my biggest single shareholding in my portfolio. Its Phase 3 data on XLRP should come out real soon now. I really love its overall company profile. And the drug really is saving a patient's life. Both Sanofi and JnJ are it's big shareholders. This is very rare.
I will not give my detailed due diligence result here. But this gene therapy company is something you want to follow or even invest.
Korean researchers figured out a way to throw a few genetic switches to cause cancer cells to revert back to a healthy state.
https://newatlas.com/cancer/cancer-cells-normal/ #Cancer #Research #Biology #Medicine #CancerResearch #GeneTherapy
Could a rare gene delay Alzheimer’s? APOE3 Christchurch inspires hope for future therapies https://www.psypost.org/could-a-rare-gene-delay-alzheimers-apoe3-christchurch-inspires-hope-for-future-therapies/?utm_source=dlvr.it&utm_medium=mastodon #Alzheimers #GeneTherapy #HealthResearch #APOE3 #Neuroscience
DATE: December 18, 2024 at 07:30AM
SOURCE: BioWorld MedTech
Direct article link at end of text block below.
Experimental mRNA gene therapy suppresses preeclampsia in mice
#medtech #genetherapy #mRNA #preeclampsia
Here are any URLs found in the article text:
Articles can be found by scrolling down the page at https://www.bioworld.com/topics/85-bioworld-medtech .
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