article thumbnail

Gene editing: beyond the hype

pharmaphorum

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. billion in funding.

article thumbnail

Combining the Powers of Single Cell Sequencing and AI in Understanding Disease Biology and Drug Development

XTalks

The detailed complexity of these processes can be captured by creating models that combine correlates of gene and protein expression, providing insight into the molecular composition of tissues. Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Pharma Marketing Network

those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e.,

article thumbnail

Biologics – The Next Step in Revolutionary Medication

Roots Analysis

Expression Systems for Biologics The processes associated with the manufacturing of biopharmaceuticals are complex and require highly sterile and aseptic conditions. This can be attributed to the fact that the production of biopharmaceuticals requires living expression systems.

article thumbnail

Gene Switch: A Novel Platform for Switching Genes On and Off

Roots Analysis

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

Gene 40
article thumbnail

Cell-penetrating peptides as a delivery system for oligonucleotides

Drug Discovery World

Currently, only a small number of CPP-based oligonucleotide delivery examples have been reported for in vivo application. 3 positive human cells and silence the targeted genes. Unfortunately, the particles formed though this method are unstable in physiological fluids. The table below includes some of these examples. Nanoparticles 6.

article thumbnail

Delivering on the promise of gene editing

Drug Discovery World

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.