Gene Therapy

Scientific journal providing peer-reviewed research and commentaries on gene and cell therapies

[Gene Therapy @GeneTherapy_SN]

A new benchtop ES-DMA instrument sizes individual AAV9 capsids precisely enough to track their mass, matching the mass spectrometry gold standard, and resolve empty, partial, full, and overfilled particles. #GeneTherapy #AAV #OpenAccess

🔗https://t.co/dQNMwD3w76

[Gene Therapy @GeneTherapy_SN]

New review of ZFNs, TALENs, CRISPR-Cas9, base & prime editors across 9 disease areas argues clinical success hinges on matching editing mechanism to disease biology, not one default platform. #GeneTherapy #GeneEditing #CRISPR
🔗https://t.co/05PBsxnG3Y

[Gene Therapy @GeneTherapy_SN]

An #AIdesigned, lung-specific #AAV promoter drove ~13x more lung expression than the standard CASI promoter and stayed silent in the liver, across 3 delivery routes and 2 AAV capsids in a preclinical model. #RareDisease #GeneTherapy
🔗 https://t.co/7Zmu1PIkKi

[Gene Therapy @GeneTherapy_SN]

AAV gene therapy shows promise in #AlphaMannosidosis model. Treatment reduced storage lesions, extended survival and slowed disease progression with clinical benefit observed even in advanced disease. #RareDiseases #AAV #GeneTherapy
🔗https://t.co/6yBDDCHk8e

[Gene Therapy @GeneTherapy_SN]

New review focused on AOC therapeutics delves into target selection and antibody engineering principles; analyzes formats and pharmacokinetics; explores delivery efficiency; examines promise and challenges. #AOC #GeneTherapy
🔗https://t.co/tpfZS7edGt

[Gene Therapy @GeneTherapy_SN]

What if gene therapy payments worked like a mortgage—spread over 30 years, contingent on the therapy continuing to work? Simulation shows this model could align incentives for patients, payers & developers. #GeneTherapy #HealthPolicy #Innovation
🔗https://t.co/LwSAglY6Fl

[Gene Therapy @GeneTherapy_SN]

One gene therapy can cost over $2 million. Who pays? Research in @GeneTherapy_SN models 30-year performance-based annuities - payers only pay if the therapy keeps working. #GeneTherapy #RareDisease #PrecisionMedicine
🔗 https://t.co/LwSAglY6Fl

[Gene Therapy @GeneTherapy_SN]

Spatial transcriptomics + snRNA-seq reveal rAAV2 favours periportal and rAAV9 pericentral hepatocytes in mouse liver — with sex-specific transduction differences. Key data for rational serotype selection. #GeneTherapy #AAVTherapy
🔗https://t.co/08s6hzUZMZ

[Gene Therapy @GeneTherapy_SN]

Methylmalonic acidemia (MMA) is a life-threatening metabolic disorder. First-in-human Phase 1/2 trial data show gene editing can safely deliver a functional enzyme in children — without cutting DNA. #GeneTherapy #RareDisease #GeneticMedicine
🔗https://t.co/GrZYXjIEUY

[Gene Therapy @GeneTherapy_SN]

Mapping the #AAV gene-therapy landscape in #nAMD: how delivery route shapes target access, dosing strategy, immune/antibody constraints, and inflammation risk—plus what today’s clinical programs are testing.
🔗 https://t.co/i4tH4kHEv3

#Retina #Ophthalmology

[Gene Therapy @GeneTherapy_SN]

Baboon ERV (BaEV)–pseudotyped lentiviral vectors outperform human HERV-W envelopes for #genedelivery into primary human cells and HSPCs and show markedly higher transduction of human SCID-repopulating cells.

🔗 https://t.co/tHQb3yAE5b
#GeneTherapy #HSPC #Biomanufacturing

[Gene Therapy @GeneTherapy_SN]

An outpatient-oriented drinkable gene therapy foam coats the esophagus, forming a local reservoir for sustained mRNA-LNP delivery—demonstrates superiority over liquid suspension in vitro.
🔗 https://t.co/Ag3oRGuRNZ

#DrinkableGeneTherapy #OesophagealCancer #DrugDelivery

[Gene Therapy @GeneTherapy_SN]

In PDAC models, patient-derived CIKs carrying CXCR3/CCR5 respond to CXCL10/CCL5. Local, drug-controlled IL-12 boosts these chemokines driving strong antitumor effects-mainly by recruiting host lymphocytes.
🔗https://t.co/wRmUB64qac
#GeneTherapy #ImmunoOncology #PancreaticCancer

[Gene Therapy @GeneTherapy_SN]

Across 10 NHP studies, AAV9 expression efficiency favored CNS over liver independent of administration route, age, or construct. Data supports epigenetic silencing as a key liver barrier.
🔗https://t.co/MRvUuCNIWN

#AAV9 #NHP #GeneTherapy #Genomics

[Gene Therapy @GeneTherapy_SN]

A streamlined, non-viral #CRISPR platform for CD34⁺ #HSPCs: efficient RNP delivery, preserved viability, rapid assembly <2 h, and low material cost (~$70/1M cells)

#ResearchInnovation #CellTherapy #ExVivoEditing
🔗 https://t.co/lM0FUDy6FF

[Gene Therapy @GeneTherapy_SN]

Single-vector #AAV packs a multi-target #ZFN pair cutting 86 Snord115 repeats in long non-coding antisense RNA, Ube3a-ATS, durably switching ON paternal UBE3A in #AngelmanSyndrome models—while sparing #PraderWilliSyndromelinked genes

🔗https://t.co/0kdexiyAb4

[Gene Therapy @GeneTherapy_SN]

What does it take to deliver CRISPR to the right tissue? This review explains rAAV pros/cons, durability, and what vector size means for safety and efficacy.
🔗 https://t.co/OgheIAPqct

#GeneTherapy #CRISPR #AAV #TranslationalMedicine

[Gene Therapy @GeneTherapy_SN]

Allele-selective AAV-RNAi for GNAO1 encephalopathy boosts WT:mutant GNAO1 (~3.8×) in patient neurons. However, transcriptomics reveal treatment-induced toxicity signals.
📖 https://t.co/C1UxTRLf5D
#Neurogenetics #RNAi #AAV #RareDisease #Transcriptomics

[Gene Therapy @GeneTherapy_SN]

A minimally invasive ultrasound-guided AAV9 delivery in utero shows safe, widespread gene transfer—no toxicity, minimal immune response, strong translation potential for severe monogenic and mitochondrial diseases.
📖https://t.co/njImzD1Avr

#GeneTherapy #FetalMedicine #AAV

[Gene Therapy @GeneTherapy_SN]

Gene therapy doesn’t end at infusion. This review of 103 long-term follow-up studies on https://t.co/Bz4d9BLnwc shows wide variation in safety monitoring and adverse-event terms—making comparisons difficult.
📖https://t.co/t6g58TkGot

#GeneTherapy #PatientSafety #LongTermFollowUp