#genetherapy — Public Fediverse posts

Adenine base editing, a highly targeted form of genetic medicine, has been successfully deployed in a preclinical mouse model to correct the specific DNA mutation (SCN1A) responsible for Dravet syndrome, a severe and often fatal form of childhood epilepsy.
#MolecularGenetics #Neurobiology #GeneTherapy #PrecisionMedicine #sflorg
https://www.sflorg.com/2026/05/gen05142601.html

🧠 Could the brain reveal vision loss more accurately than traditional eye tests?

🔗 Using Steady-State Visual Evoked Potentials to Characterize Wide-Ranging Retinopathy Linked to CRB1: Implications for Clinical Trials. Computational and Structural Biotechnology Journal (CSBJ). DOI: https://doi.org/10.34133/csbj.0042

📚 CSBJ - A Science Partner Journal: https://spj.science.org/journal/csbj

#Neuroscience #Ophthalmology #RetinalDiseases #GeneTherapy #EEG #BrainResearch #DigitalHealth #PrecisionMedicine #Neuroimaging

🧠 Could the brain reveal vision loss more accurately than traditional eye tests?

🔗 Using Steady-State Visual Evoked Potentials to Characterize Wide-Ranging Retinopathy Linked to CRB1: Implications for Clinical Trials. Computational and Structural Biotechnology Journal (CSBJ). DOI: https://doi.org/10.34133/csbj.0042

📚 CSBJ - A Science Partner Journal: https://spj.science.org/journal/csbj

#Neuroscience #Ophthalmology #RetinalDiseases #GeneTherapy #EEG #BrainResearch #DigitalHealth #PrecisionMedicine #Neuroimaging

🧠 Could the brain reveal vision loss more accurately than traditional eye tests?

🔗 Using Steady-State Visual Evoked Potentials to Characterize Wide-Ranging Retinopathy Linked to CRB1: Implications for Clinical Trials. Computational and Structural Biotechnology Journal (CSBJ). DOI: https://doi.org/10.34133/csbj.0042

📚 CSBJ - A Science Partner Journal: https://spj.science.org/journal/csbj

#Neuroscience #Ophthalmology #RetinalDiseases #GeneTherapy #EEG #BrainResearch #DigitalHealth #PrecisionMedicine #Neuroimaging

🧠 Could the brain reveal vision loss more accurately than traditional eye tests?

🔗 Using Steady-State Visual Evoked Potentials to Characterize Wide-Ranging Retinopathy Linked to CRB1: Implications for Clinical Trials. Computational and Structural Biotechnology Journal (CSBJ). DOI: https://doi.org/10.34133/csbj.0042

📚 CSBJ - A Science Partner Journal: https://spj.science.org/journal/csbj

#Neuroscience #Ophthalmology #RetinalDiseases #GeneTherapy #EEG #BrainResearch #DigitalHealth #PrecisionMedicine #Neuroimaging

🧠 Could the brain reveal vision loss more accurately than traditional eye tests?

🔗 Using Steady-State Visual Evoked Potentials to Characterize Wide-Ranging Retinopathy Linked to CRB1: Implications for Clinical Trials. Computational and Structural Biotechnology Journal (CSBJ). DOI: https://doi.org/10.34133/csbj.0042

📚 CSBJ - A Science Partner Journal: https://spj.science.org/journal/csbj

#Neuroscience #Ophthalmology #RetinalDiseases #GeneTherapy #EEG #BrainResearch #DigitalHealth #PrecisionMedicine #Neuroimaging

This story is spreading because it shows how close we are to curing diseases at their root.
#GeneTherapy #Healthcare #Science #MedicalInnovation #GlobalHealth #Biotech

👂🧬 The #FDA approved #Otarmeni, the first gene therapy designed to restore #hearing in individuals with a rare form of genetic #deafness caused by OTOF gene mutations.

The one-time treatment delivers a functional copy of the gene into the inner ear, enabling cells to transmit #sound signals to the #brain.

👉 https://www.livescience.com/health/genetics/a-landmark-moment-for-the-field-fda-approves-first-ever-gene-therapy-for-inherited-deafness

#genetics #health #science #biology #medicine #biotechnology #research #genetherapy #innovation

FDA Approves First-Ever Gene Therapy for Treatment of Genetic Hearing Loss

https://www.fda.gov/news-events/press-announcements/fda-approves-first-ever-gene-therapy-treatment-genetic-hearing-loss-under-national-priority-voucher

#HackerNews #FDA #GeneTherapy #HearingLoss #MedicalInnovation #GeneticResearch

Six-Year-Old's Vision Altered by Gene Intervention

Six-year-old Saffie Sandford from Stevenage sees better after Luxturna gene therapy for Leber's Congenital Amaurosis at Great Ormond Street Hospital.

#GeneTherapy, #Luxturna, #LCA, #Stevenage, #EyeHealth

https://newsletter.tf/stevenage-girl-vision-gene-therapy-luxturna-lca/

A six-year-old girl's vision has improved significantly after a one-off gene therapy treatment. This is the first time this therapy has been used on such a young child for this condition.

#GeneTherapy, #Luxturna, #LCA, #Stevenage, #EyeHealth
https://newsletter.tf/stevenage-girl-vision-gene-therapy-luxturna-lca/

Common gene in axolotls, mice and zebrafish may unlock human limb regrowth

Investigating a common gene in three very different species – axolotls, mice and zebrafish – scientists have discovered…
#NewsBeep #News #Health #Amputations #AU #Australia #Bone #Brain #Cancer #diabetes #Epidermis #Gene #genetherapy #Genes #Genetic #Heart #research #Skin #therapy #Vascular
https://www.newsbeep.com/au/622119/

Common gene in axolotls, mice and zebrafish may unlock human limb regrowth

Investigating a common gene in three very different species – axolotls, mice and zebrafish – scientists have discovered…
#NewsBeep #News #Health #Amputations #AU #Australia #Bone #Brain #Cancer #diabetes #Epidermis #Gene #genetherapy #Genes #Genetic #Heart #research #Skin #therapy #Vascular
https://www.newsbeep.com/au/622119/

‘Oscar of science’ awarded to team behind gene therapy that restores lost vision
https://www.theguardian.com/science/2026/apr/19/the-oscar-of-science-awarded-to-scientists-behind-genetic-treatment-that-restores-lost-vision-win
#news #science #GeneTherapy #breakthrough #blindness #vision

https://www.europesays.com/ch/51103/ Roche plots route to EMA approval for DMD gene therapy #BioWorld #ChugaiPharmaceuticalCoLtd #DuchenneMuscularDystrophy #Elevidys #EMA #Europe #GeneTherapy #JAPAN #Musculoskeletal #Regulatory #Roche #RocheAG #SareptaTherapeuticsInc

Shionogi makes investment in Japanese startup Restore Vision to develop visual restoration gene therapy

Shionogi makes investment in Japanese startup Restore Vision to develop visual restoration gene therapy April 16, 2026 | Thursday | News Shionogi’s first investment under its…
#Japan #JP #JapanNews #genetherapy #invest #Japanese #Japanesenews #news #startup
https://www.alojapan.com/1476213/shionogi-makes-investment-in-japanese-startup-restore-vision-to-develop-visual-restoration-gene-therapy/

Shionogi makes investment in Japanese startup Restore Vision to develop visual restoration gene therapy

Shionogi makes investment in Japanese startup Restore Vision to develop visual restoration gene therapy April 16, 2026 | Thursday | News Shionogi’s first investment under its…
#Japan #JP #JapanNews #genetherapy #invest #Japanese #Japanesenews #news #startup
https://www.alojapan.com/1476213/shionogi-makes-investment-in-japanese-startup-restore-vision-to-develop-visual-restoration-gene-therapy/

Woman with three deadly diseases has ‘remarkable’ recovery after cell therapy;
Treatment reset wayward immune system of patient with life-threatening conditions, say scientists, in a world first
https://www.theguardian.com/science/2026/apr/09/autoimmune-diseases-cell-therapy-immune-reset
#science #news #medicine #GeneTherapy #health #breakthrough

11-Mar-2026
Researchers develop new way to safely insert #gene-sized #DNA into the #genome

https://www.eurekalert.org/news-releases/1119454 #science #geneTherapy

Abeona Therapeutics® Provides Business Update Highlighting Building Momentum in ZEVASKYN® Commercial Launch

Abeona Therapeutics Inc. CLEVELAND, March 09, 2026 (GLOBE NEWSWIRE) — Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced an…
#UnitedStates #US #USA #AbeonaTherapeuticsInc. #america #business #genetherapy #pediatricpatients #RDEB #unitedstatesofamerica #ZEVASKYN
https://www.europesays.com/2833377/

Two-decade trends in prenatal genetic testing in Japan https://www.byteseu.com/1840397/ #GeneExpression #GeneFunction #GeneTherapy #GeneticTesting #GeneticsResearch #HumanGenetics #Japan #MolecularMedicine

Gene therapy revolutionizes medicine by correcting defective genes and treating inherited diseases, cancers, and rare disorders with advanced biotechnology innovations.
#GeneTherapy #Biotechnology #GeneticEngineering #MedicalInnovation #PrecisionMedicine #CancerResearch #RareDiseases #BiotechStudents #LifeSciences #BioResire

Gene therapy revolutionizes medicine by correcting defective genes and treating inherited diseases, cancers, and rare disorders with advanced biotechnology innovations.
#GeneTherapy #Biotechnology #GeneticEngineering #MedicalInnovation #PrecisionMedicine #CancerResearch #RareDiseases #BiotechStudents #LifeSciences #BioResire

https://www.europesays.com/ie/348278/ Genetic mismatch identified as major risk factor in cord blood transplantation #Antigen #blood #Bone #BoneMarrow #Cell #education #Éire #Gene #GeneTherapy #Genetic #Health #HumanLeukocyteAntigen #IE #Ireland #Leukocyte #medicine #Q #Research #StemCells #Students #therapy #Transplant #UmbilicalCord

Gene therapy transforms medicine by correcting defective genes, offering hope for genetic disorders, cancer treatment, and future precision healthcare breakthroughs.
#GeneTherapy #Biotechnology #GeneticEngineering #PrecisionMedicine #MolecularBiology #BiotechInnovation #MedicalScience #LifeSciences #FutureMedicine #HealthcareInnovation

Gene therapy transforms medicine by correcting defective genes, offering hope for genetic disorders, cancer treatment, and future precision healthcare breakthroughs.
#GeneTherapy #Biotechnology #GeneticEngineering #PrecisionMedicine #MolecularBiology #BiotechInnovation #MedicalScience #LifeSciences #FutureMedicine #HealthcareInnovation

Gene therapy transforms medicine by correcting defective genes, offering hope for genetic disorders, cancer treatment, and future precision healthcare breakthroughs.
#GeneTherapy #Biotechnology #GeneticEngineering #PrecisionMedicine #MolecularBiology #BiotechInnovation #MedicalScience #LifeSciences #FutureMedicine #HealthcareInnovation

Gene therapy transforms medicine by correcting defective genes, offering hope for genetic disorders, cancer treatment, and future precision healthcare breakthroughs.
#GeneTherapy #Biotechnology #GeneticEngineering #PrecisionMedicine #MolecularBiology #BiotechInnovation #MedicalScience #LifeSciences #FutureMedicine #HealthcareInnovation

Gene therapy transforms medicine by correcting defective genes, offering hope for genetic disorders, cancer treatment, and future precision healthcare breakthroughs.
#GeneTherapy #Biotechnology #GeneticEngineering #PrecisionMedicine #MolecularBiology #BiotechInnovation #MedicalScience #LifeSciences #FutureMedicine #HealthcareInnovation

Lipid #nanoparticles successfully delivered a full-length, healthy #CFTR gene into human airway cells, restoring essential biological function in a cystic #fibrosis model without the use of viral vectors.
#Medical #Nanotechnology #Nanomedicine #GeneTherapy #PulmonaryMedicine #sflorg
https://www.sflorg.com/2026/02/med02172601.html

Discover gene therapy fundamentals, vectors, and applications transforming modern medicine and biotechnology with BioResire’s quick learning guide.
#GeneTherapy #Biotechnology #GeneticEngineering #PrecisionMedicine #CancerTherapy #Genomics #MolecularMedicine #CRISPR #LifeSciences #BioResire

Discover gene therapy fundamentals, vectors, and applications transforming modern medicine and biotechnology with BioResire’s quick learning guide.
#GeneTherapy #Biotechnology #GeneticEngineering #PrecisionMedicine #CancerTherapy #Genomics #MolecularMedicine #CRISPR #LifeSciences #BioResire

Discover gene therapy fundamentals, vectors, and applications transforming modern medicine and biotechnology with BioResire’s quick learning guide.
#GeneTherapy #Biotechnology #GeneticEngineering #PrecisionMedicine #CancerTherapy #Genomics #MolecularMedicine #CRISPR #LifeSciences #BioResire

Boy with rare condition amazes doctors after #worldfirst #genetherapy

https://www.bbc.com/news/articles/c5y0y56x6veo?&at_bbc_team=studios

Boy with rare condition amazes doctors after #worldfirst #genetherapy

https://www.bbc.com/news/articles/c5y0y56x6veo?&at_bbc_team=studios

Boy with rare condition amazes doctors after #worldfirst #genetherapy

https://www.bbc.com/news/articles/c5y0y56x6veo?&at_bbc_team=studios

Boy with rare condition amazes doctors after #worldfirst #genetherapy

https://www.bbc.com/news/articles/c5y0y56x6veo?&at_bbc_team=studios

🧬 Can controlling PARP1 turn CRISPR from a gene scissor into a gene sculptor?

🔗 Influence of PARP1 on CRISPR/Cas9 induced double strand break repair in proliferating cells. Computational and Structural Biotechnology Journal, DOI: https://doi.org/10.1016/j.csbj.2025.10.001

📚 CSBJ: https://www.csbj.org/

#CRISPR #GenomeEditing #Genomics #DNARepair #PARP1 #GeneticEngineering #MolecularBiology #GeneTherapy #CellBiology #SyntheticBiology #TherapeuticEditing

Opus Genetics’ BEST1 Gene Therapy IND Cleared by FDA

Opus Genetics (Nasdaq: IRD) has received FDA clearance for its…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Genetics #AAVtherapy #Bestdisease #BEST1 #FDAclearance #GeneTherapy #GeorgeMagrath #INDapplication #inheritedretinaldisease #IRD #OPGx-BEST1 #OpusGenetics #Phase1/2trial #Science #subretinalinjection #vitelliformmaculardystrophy
https://www.newsbeep.com/us/91789/

Opus Genetics’ BEST1 Gene Therapy IND Cleared by FDA

Opus Genetics (Nasdaq: IRD) has received FDA clearance for its…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Genetics #AAVtherapy #Bestdisease #BEST1 #FDAclearance #GeneTherapy #GeorgeMagrath #INDapplication #inheritedretinaldisease #IRD #OPGx-BEST1 #OpusGenetics #Phase1/2trial #Science #subretinalinjection #vitelliformmaculardystrophy
https://www.newsbeep.com/us/91789/

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

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

#AAV #GeneTherapy

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

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

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

DATE: November 08, 2024 at 07:00AM
SOURCE: BioWorld MedTech

Direct article link at end of text block below.

FDA eases the way for cell and gene therapy companies

https://t.co/J3hTx0sL9y

#medtech @US_FDA #genetherapy #celltherapy

Here are any URLs found in the article text:

https://t.co/J3hTx0sL9y

#medtech

Articles can be found by scrolling down the page at https://www.bioworld.com/topics/85-bioworld-medtech .

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#healthcare #healthtech #healthcaretech #healthtechnology #medgadget #medicine #doctor #hospital #medtech

DATE: November 07, 2024 at 04:59PM
SOURCE: BioWorld MedTech

Direct article link at end of text block below.

FDA eases the way for cell and gene therapy companies

https://t.co/J3hTx0sL9y

#medtech @US_FDA #genetherapy #celltherapy

Here are any URLs found in the article text:

https://t.co/J3hTx0sL9y

#medtech

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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=86419

DATE: October 25, 2024 at 07:00AM
SOURCE: BioWorld MedTech

Direct article link at end of text block below.

ESGCT 2024: Steps forward in gene and cell therapies for brain tumors

https://t.co/M9da2SNzSw

#medtech #genetherapy #celltherapy #tumor

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#medtech

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DATE: October 24, 2024 at 04:00PM
SOURCE: BioWorld MedTech

Direct article link at end of text block below.

ESGCT 2024: Steps forward in gene and cell therapies for brain tumors

https://t.co/M9da2SO7I4

#medtech #genetherapy #celltherapy #tumor

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#medtech

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