We are proud to announce that 𝘁𝗵𝗲 𝗳𝗶𝗿𝘀𝘁 𝗽𝗮𝘁𝗶𝗲𝗻𝘁 𝗵𝗮𝘀 𝗯𝗲𝗲𝗻 𝗱𝗼𝘀𝗲𝗱 𝗶𝗻 𝘁𝗵𝗲 𝗦𝗢𝗡𝗜𝗫 𝗣𝗵𝗮𝘀𝗲 𝟭/𝟮 𝘁𝗿𝗶𝗮𝗹 𝗼𝗳 𝗦𝗞𝗬-𝗚𝗝𝗕𝟮. This important milestone advances the development of a potential one-time gene therapy for chi...

Viral Vectors 101: Systemic Capsids

The head of the U.S. Food and Drug Administration's vaccines and biologics unit, Dr. Vinay Prasad, ​will leave the agency at the end of April, ‌a spokesperson for the U.S. Department of Health and Hum...

This study reveals SKG0201, a CNS-targeted AAV9-SMN1 gene therapy, achieved long-term survival and motor improvements in SMA mice at lower dose than approved therapy. In a Phase 1 trial, infants showed functional gains and safety, supporting its enhanced therapeutic index for SMA type 1.

Adeno associated virus-based gene therapy is a revolutionary treatment method that treats otherwise untreatable genetic diseases. However, organ toxicity remains a safety concern in the clinic. This work provides unique transcriptional activities that provide evidence of the early mechanisms that drive this critical side effect in AAV-based gene therapy.

Genetic deficiency of otoferlin, a protein critical to synaptic transmission by the sensory hair cells of the ear, causes congenital deafness. Medicines to treat the condition are lacking; children...

Engineering a mutant-specific customized base editor precisely corrects a mutation while minimizing bystander edits, leading to substantial phenotypic recovery in mouse models of multisystemic smooth ...

Nature Biomedical Engineering - Engineering a mutant-specific customized base editor precisely corrects a mutation while minimizing bystander edits, leading to substantial phenotypic recovery in...

UMass Chan Medical School is rescinding admission offers to candidates for its biomedical sciences PhD program over concerns about federal funding cuts.

Everything from cancer research to the distribution of medical information is affected.

Addgene's AAV Packaged on Request option has officially launched! Learn more about how this expansion of our helpful viral vectors service can help your research.

Qiu and colleagues identified that the membrane-associated accessory protein (MAAP) of AAV is associated with the SNARE complex. They also revealed that the knockout of the SNARE proteins, STX7 or SNAP23, enhances the secretion of AAV from the cells and increases the overall vector yield during vector production in HEK293 cells.

Adeno-associated viruses (AAV) are promising vectors for gene therapy due to their efficacy in vivo. However, there is room for improvement to address…

We previously showed therapeutic advantages of using a capsid-modified and encoded antigen-optimized AAV-based cancer vaccine to initiate strong antig…

Landegger and colleagues discuss several recent clinical trials that have demonstrated gene therapy’s safety and effectiveness in treating hearing loss. While many questions remain, ongoing research a...