Adeno-associated virus (AAV) is a small, non-pathogenic, and non-enveloped virus that has become an important tool for gene therapy research and applications. AAV is a type of virus that requires a helper virus (such as adenovirus) to replicate, and it has a high degree of stability and safety, as it does not...
Therapeutics based on messenger RNA, or mRNA, can potentially treat a wide range of maladies, including cancer, genetic diseases, and as the world has learned in recent years, deadly viruses. To work, these drugs must be delivered directly to target cells in nanoscale bubbles of fat called lipid nanoparticles, or LNPs — mRNA isn’t [...]
Scientists at St. Jude Children’s Research Hospital have developed a method that may be able to improve CAR T-cell therapies by identifying the early cells that become effective at killing cancer. St. Jude Children’s Research Hospital scientists have demonstrated that the long-term performance of cancer-killing chimeric antigen receptor (CAR) T cells can, in some cases, be predicted [...]
A paper from the Horae Gene Therapy Center at UMass Chan Medical School may settle a longstanding debate about how to best manufacture adeno-associated virus (AAV) vectors for gene therapy. The paper, published online in the journal Human Gene Therapy, describes using AAV genome population sequencing (AAV-GPseq) to explore the genomes of vectors produced by two [...]
