The tetracycline-controlled Tet-Off and Tet-On gene expression systems are used to regulate
the activity of genes in eukaryotic cells in diverse settings, varying from basic biological …

Recombinant adeno-associated viruses (AAV) are under intensive investigation in
numerous clinical trials after they have emerged as a highly promising vector for human …

Synthetic gene circuits that precisely control human cell function could expand the
capabilities of gene-and cell-based therapies. However, platforms for developing circuits in …

The capacity to efficiently transduce nondividing cells, shuttle large genetic payloads, and
maintain stable long-term transgene expression are attributes that have brought lentiviral …

Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …

Genome‐editing therapeutics are poised to treat human diseases. As we enter clinical trials
with the most promising CRISPR‐Cas9 and CRISPR‐Cas12a (Cpf1) modalities, the risks …

Chondroitinase ABC is a promising preclinical therapy that promotes functional
neuroplasticity after CNS injury by degrading extracellular matrix inhibitors. Efficient delivery …

Gene therapy aims to revert diseased phenotypes by the use of both viral and nonviral gene
delivery systems. Substantial progress has been made in making gene transfer vehicles …

Gene therapy is a potential route for the delivery of secreted therapeutic proteins, but
pharmacologic control of expression will generally be required for optimal safety and …

Neurodegenerative disorders are a major public health problem because of the high
frequency of these diseases. Genome editing with the CRISPR/Cas9 system is making it …