Adeno-associated virus (AAV)-based gene therapy is a biological vector that is being researched to be used as a potential therapeutic option. This gene therapy is designed to insert fragments of DNA into targeted cells to help treat diseases, such as hemophilia a. Discover the latest research on AAV-based gene therapy here.
Antisense oligonucleotides are synthetic DNA oligomers that hybridize to a target RNA. This feed focuses on antisense oligonucleotide therapies such as Inotersen, Nusinursen, and Patisiran, in neurodegenerative diseases.
Over 1700 different mutations in the CFTR genes have been shown to cause cystic fibrosis. Here is the latest research on structural therapy for CFTR mutants.
Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.
CRISPR-Cas system enables the editing of genes to create or correct mutations. This feed focuses on the application of this system for gene editing and therapy in human diseases.
The use of DNA as a technique for treating disease is a key feature of gene therapy. This type of treatment is being investigated to either replacement of a mutated gene with a healthy copy, silence a mutated gene that is dysfunctional or insert a novel gene to help fight disease. Discover the latest research on gene therapy here.
Gene therapy is emerging as a potential strategy for the treatment of cardiovascular diseases, such as peripheral arterial disease, ischemic heart disease, restenosis after angioplasty, vascular bypass graft occlusion and transplant-associated coronary artery disease. Discover the latest research on gene therapy for cardiovascular diseases here.
Gene therapy uses genes to treat or prevent diseases such as muscle diseases. Here are the latest discoveries pertaining to gene therapy and muscle diseases.
Genetic engineering is a process that alters the genetic structure of an organism by removing or introducing DNA. This technique is being tested to program cells. Discover the latest research on genetic engineering to program cells here.
This feed focuses on the high throughput technologies used to dissect the genetics of behavior and their associated disorders. Discover the latest research here.
Genome editing technologies enable the editing of genes to create or correct mutations or express genes of interest. Here is the latest research on genome editing in T- cells and their application in human diseases such as adoptive T-cell immunotherapy for cancer.
Recent developments in genomics and further development of advanced post-genomic technologies, it will be possible to apply a more holistic analysis to genomic analysis. Discover the latest research in Genomic Techniques.
This feed focuses on emerging cell and gene therapies, such as HIV vaccines, for treating patients living with HIV/AIDS. Discover the latest research here.
Mixed lineage leukemia-1 (MLL-1) is a particular type of acute leukemia from the mutation of the MLL/KMT2A gene. This mutation can be seen in pediatrics, adults and therapy-induced acute leukemia’s. Discover the latest research on MLL-r leukemia here.
Gene therapy is a powerful tool that can potentially be used for treating neurodegenerative diseases such as amyotrophic lateral sclerosis, spinal muscular atrophy, Parkinson's disease, and Alzheimer's disease. Follow the latest research in this field with this feed.
Gene therapy and protein engineering strategies, guided by biological principles, are allowing ligands and receptors to be developed as next-generation therapeutics with improved safety and efficacy. Discover the latest research here.
Transcription activator-like effectors (TALEs) and TALE nucleases (TALENs) enable gene editing by targeting specific DNA sequences in the genome. Here is the latest research.
The delivery of genes into DNA for gene therapy is done through the use of vectors, which are often viral vectors. These vectors can be manipulated to include DNA and RNA depending on the type of therapy that is needed. Here is the latest research on vector gene therapy.