Learn more about the current status of AAV gene therapy as a potential treatment for GM1 gangliosidosis.

READ: curegm1.org/aav-gene-therapy-for-gm1

#curegm1 #raredisease #aav #genetherapy #research

Excited to share the work from my PhD looking into the mechanism of #AAV 🦠 packaging using #CryoEM ❄️🔬 is out now:
pubmed.ncbi.nlm.nih.gov/41790558/

Massive thanks to my supervisors Sarah Rouse, @doryenbubeck.bsky.social and Matt Wake!

Manufacturing contaminants linked with toxicity in gene therapies A research team from London examined the relationship between adenovirus-associated virus (AAV)-based vectors and liver toxicity in gene therapy patients.

AAV-associated toxicity in gene therapies could be caused by contaminants in the manufacturing process, a research team based out of London claim. Liver biopsies tested positive for viral fragments.

Read my full article below:
#genetherapy #AAV #news

www.bioprocessintl.com/therapeutic-...

Fluorescent “superheroes”！ mBaoJin + mScarlet3-H (also known as mYongHong) Tool Viruses Empower Super-Resolution and High-Efficiency Imaging Discover Brain Case Biotech's viral vectors with mBaoJin and mScarlet3-H proteins, offering superior photostability and precise delivery for advanced imaging in cell biology and neuroscience.

🎉 Fluorescent “superheroes”！
📍Tired of photobleaching and weak signals limiting your imaging?
Explore the full product list, from synaptic labeling to mitochondrial markers, via the link below!
www.ebraincase.com/news/new-pro...

#Neuroscience #CellBiology #SuperResolution #FluorescentProtein #AAV

Text which says "Scaling AAV Manufacturing for Neuromuscular Gene Therapy" alongside an image on an AAV

AAV gene therapies can only progress if we can manufacture high‑quality vectors at scale.

MAGIC and DINAMIQS have been working to make AAV production more robust and development‑ready, helping ensure emerging neuromuscular therapies have a practical path toward the clinic.

#GeneTherapy #AAV

国産HAT細胞活用による高品質AAV製造技術の確立 国産HAT細胞を用いた高生産性・高品質なAAV製造プラットフォームが確立。遺伝子治療薬の供給が期待される最新の研究結果をご紹介します。

国産HAT細胞活用による高品質AAV製造技術の確立 #大阪府 #大阪市 #遺伝子治療 #AAV #HAT細胞

国産HAT細胞を用いた高生産性・高品質なAAV製造プラットフォームが確立。遺伝子治療薬の供給が期待される最新の研究結果をご紹介します。

Graphical abstract illustrating cancer stem cell (CSC) heterogeneity driven by genetic, epigenetic, and environmental factors, and a proposed miRNA-dependent AAV strategy selectively eradicating heterogeneous CSCs to prevent recurrence.

📘 CHP Archive Spotlight #06
Perspective (2024) | Kondo
Targeting cancer stem cell heterogeneity & plasticity with a miRNA-dependent AAV system designed to eradicate diverse CSC populations while minimizing side effects.
🔗 doi.org/10.47248/chp...
#CancerStemCells #Heterogeneity #AAV

🧬 AAV product discounts now available from selected 2BScientific suppliers.

Support your gene therapy & viral vector research with high-quality AAV tools at reduced pricing.

📩 Contact us for details & quotes.
buff.ly/CQhqTVO

#AAV #GeneTherapy #ViralVectors #LifeScience

Pulmonary Manifestations Affect 4 in 5 Individuals With AAV Pulmonary involvement is highly prevalent in AAV, affecting more than 80% of patients with GPA and nearly 75% of those with MPA.

Pulmonary involvement is highly prevalent in individuals with #ANCA-associated vasculitis (#AAV), affecting 82.7% of patients with granulomatosis with polyangiitis and 74.1% of those with microscopic polyangiitis.

Read more: https://bit.ly/4aXj0Zv

#RareDisease #MPA #GPA #Rheumatology

#ILD 🫁 and #ANCA #AAV 👌

It was great to have our former postdoc Helena Costa-Verdera visit us @unipv.bsky.social today. Her work on #AAV signaling in the CNS continues 🧠🦠🧬 Stay tuned!

Meet us at #AdvancedTherapiesWeek in San Diego today at booth 456. Get what other vector design and manufacturing services can’t give you: fast turnaround times, consistently low endotoxin levels, and dedicated technical support. #ATW2026 #GeneTherapy #AAV #Adenovirus #shRNA

Early discovery is exciting. Building the translational backbone is the hard part. Today at #AdvancedTherapiesWeek in San Diego: fireside chat on translational infrastructure—connecting discovery to clinical needs with fit-for-purpose workflows + more predictive systems. #ATW2026 #GeneTherapy #AAV

Meet us at #AdvancedTherapiesWeek in San Diego, Feb 9–12, at booth 456. Get what other vector design and manufacturing services can’t give you: fast turnaround times, consistently low endotoxin levels, and dedicated technical support. #ATW2026 #GeneTherapy #AAV #Adenovirus #shRNA

How to use Targeted Viral Vectors for Disease Modeling & Therapeutic Discovery The ability to deliver exogenous genes in targeted viral vectors for disease modeling is revolutionizing therapeutic discovery. (Wang et al. 2024, Zwi-Dantsis et al. 2025, Haggerty et al. 2019, Naso…

Build better disease models with targeted viral vectors—AAV + adenovirus design guidance. #GeneTherapy #AAV #Adenovirus #shRNA tinyurl.com/3adcfk5r

Early discovery is exciting. Building the translational backbone is the hard part. Feb 10 at #AdvancedTherapiesWeek in San Diego: fireside chat on translational infrastructure—connecting discovery to clinical needs with fit-for-purpose workflows + more predictive systems. #ATW2026 #GeneTherapy #AAV

Rational engineering of the P5 TRS-mimic site and REP78/68 start codon yields promoter variants that improve rAAV vector purity while maintaining high titers. During recombinant adeno-associated virus (rAAV) production, certain components of the manufacturing system can be encapsidated as unwanted nucleic acid contaminants. Prior work has established that t...

Congratulations to now graduated PhD student Stephen Winston for leading this study on rAAV contaminants.

This study shows that a mere 2 nucleotide modification to the (Rep78/68 driving) p5 promoter results in rAAV with equivalent titers but ~50-fold lower p5-derived contaminants. #genetherapy #AAV

Patient With AAV Later Diagnosed With IgA Nephropathy An elderly patient with ANCA-associated vasculitis was later diagnosed with IgA nephropathy following a repeat kidney biopsy.

An elderly patient with antineutrophil cytoplasmic antibody (#ANCA)-associated vasculitis (#AAV) had repeated #KidneyBiopsies showing new-onset IgA nephropathy. Study in CEN Case Reports.

Read more: https://bit.ly/4btyZQW

#RareDisease #ANCAAssociatedVasculitis #MedSky

AAV vs. Lentivirus: Key Differences and Applications of Two Masterpieces in Gene Delivery A comprehensive comparison of AAV and lentiviral vectors, covering gene delivery mechanisms, packaging capacity, immunogenicity, and in vivo vs in vitro applications, with case studies from gene thera...

🧬 #AAV vs. #Lentivirus: How do you choose the right #genedelivery vector?

This article shows their key differences, ideal use cases, and examples to help you decide which #vector fits your project best.

🔗 www.ebraincase.com/support/lite...

#GeneDelivery #ViralVectors #GeneTherapy #Neuroscience

Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration | Human Gene Therapy CLN5 disease, caused by mutations in the CLN5 gene, is a form of neuronal ceroid lipofuscinoses (Batten disease). Patients suffer progressive motor dysfunction, vision loss, seizures, and dementia, leading to premature death. Here, we report a preclinical study of AAV9-mediated gene therapy in a Cln5−/− mouse model. Single-dose AAV9 carrying human CLN5 driven by the CAG or human synapsin 1 promoter (hSYN) was administered via intracerebroventricular injection into neonatal and juvenile Cln5−/− mice. Treatment efficacy was evaluated by assessment of neurodegeneration, neuroinflammation, locomotor function, and survival. AAV9 expressing CLN5 driven by the hSYN promoter significantly alleviated neurodegeneration, improved biochemical and glycosphingolipid profiles, neuropathological and locomotor function, and extended lifespan of the Cln5−/− mice. However, gene transfer employing the CAG promoter demonstrated limited therapeutic efficacy. Furthermore, delayed intervention in juveniles provided superior therapeutic response compared with early neonatal intervention and normalized lifespan. Finally, blood plasma neurofilament light that is significantly elevated in the Cln5−/− mice is restored to normal wildtype levels following treatment. These results indicate that brain-directed adeno-associated virus (AAV) gene therapy could be a promising treatment strategy for CLN5 disease and efficacy might be monitored using a noninvasive blood plasma biomarker.

New paper supporting #AAV -mediated #genetherapy for neurodegenerative disease, #CLN5 neuronal ceroid lipofuscinosis. Improved neuropathology, lifespan, locomotor function, biochemistry and normalisation of plasma biomarker. @asgct.bsky.social @bsgct.bsky.social www.liebertpub.com/doi/10.1177/...

Meet us at #AdvancedTherapiesWeek in San Diego, Feb 9–12, at booth 456. Get what other vector design and manufacturing services can’t give you: fast turnaround times, consistently low endotoxin levels, and dedicated technical support. #ATW2026 #GeneTherapy #AAV #Adenovirus #shRNA

Case Report: Concurrent Ulcerative Colitis and AAV A patient with a history of ulcerative colitis was subsequently diagnosed with ANCA-associated vasculitis and was successfully treated.

A male patient with existing #UlcerativeColitis was subsequently diagnosed with antineutrophil cytoplasmic antibody (#ANCA)-associated vasculitis (#AAV) and received effective treatment, according to a case report published in Clinical Rheumatology.

Read more: https://bit.ly/45IDfYW

#Rheumatology

AAV vectors for basic research: an asprosin hormone case study How AAV vectors and a newly identified hormone connected basic mechanism to translational relevance in metabolic and neural regulation. AAV vectors for basic research have become a versatile tool to…

Learn how AAV functionality can drive translational innovation. In this story, AAV vectors + a newly identified hormone come together showing how the right vector strategy can turn foundational biology into actionable translational insight.
tinyurl.com/45y5sjpm
#GeneTherapy #AAV #Adenovirus #shRNA

UK biotech eyes $1B deal to enable eye gene therapy to be delivered in doctors' offices U.K. | U.K. biotech Ikarovec has teamed up with VectorBuilder to work on an eye disease gene therapy that could be administered in a doctor’s office.

Great 2026 news for ocular gene therapy: Ikarovec partners with VectorBuilder to attempt intravitreal delivery of IKAR-003. Nice to see continuing innovation in this challenging space. #GeneTherapy #Ophthalmology #AAV buff.ly/8Ich46b

Holiday bonus for your bench: 30% off AAV, Adenovirus & shRNA catalog vectors so your 2026 data starts strong.
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#GeneTherapy #AAV #Adenovirus #shRNA

Holiday bonus for your bench: 30% off AAV, Adenovirus & shRNA catalog vectors so your 2026 data starts strong.
AAV: tinyurl.com/3vjf27z2
Adenovirus: tinyurl.com/mtcvcdrm
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#GeneTherapy #AAV #Adenovirus #shRNA

Congrats to Dr. Pallotti & Prof. @terrierben.bsky.social on their collaborative work exploring the overlapping forms of #GPA and #EGPA, made possible through European teamwork! 🎉 #Research #Collaboration #GFEV #AAV

In-Depth Analysis of Critical Quality Attributes (CQAs) for AAV Explore the critical quality attributes (CQA) of AAV vectors, including identity, potency, purity, impurities, and safety, for safe and effective gene therapy.

✨AAV Critical Quality Attributes (CQAs) Explained🤩
AAV quality drives gene therapy safety & efficacy. This review breaks down the 5 key CQAs—identity, titer & potency, purity, impurities, and safety.

Read More👉: www.ebraincase.com/support/lite...

#GeneTherapy #AAV #Biotech #ViralVector #CQA

The abstract flowing waves depict the epileptic brain activity that is quieted by the adeno-associated virus (AAV) delivery of the SYNGAP1 gene. Overlaid on the waves to the left are EEG traces from an untreated Syngap1 mouse model, while the trace to the right of the AAV reflects the treated state. This highlights how the gene therapy developed by Levi and colleagues can stabilize disrupted neural signaling and restore balance to the brain. Image credit: Meagan Quinlan (concept, design, illustration) and Rong Guo (EEG traces).

The December 2025 issue of Molecular Therapy is out now and features new research on #AAV SYNGAP1 #GeneTherapy, #CAR-NKT cells for glioblastoma, in vivo #GeneEditing for hearing loss, and more.

Read it today: http://dlvr.it/TPdTMQ

Pick your perfect knockdown: 100K+ pre-made AAV, Adenovirus & shRNA vectors with >95% purity, low bioburden/endotoxin, and broad serotype/promoter options.
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#GeneTherapy #AAV #Adenovirus #shRNA