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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.

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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.

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Using gene therapy to live longer, healthier lives

Drug Discovery World

Our lead programme is a gene therapy called ER-100 which allows for expression of three transcription factors Oct-4, Sox-2, and Klf-4 (OSK) which is being tested in two optic neuropathies: non-arteritic anterior ischemic optic neuropathy (NAION) and primary open angle glaucoma (POAG).”

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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. It is worth mentioning that in vitro gene expression requires a suitable host for the production of a specific gene product.

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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.,

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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. CDMO in peptides and oligonucleotides manufacturing, Bachem, has started building a new future for oligonucleotides: solving the industry’s main challenges around capacity, sustainability and cost-effectiveness.

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Angelman Syndrome Market: Insights Into The Recent Late-Stage Drug Failures And Novel Approaches To Treating This Rare Neurogenetic Disorder

Delveinsight

Usually, only a single copy of the gene is required that too from the mother, however, in cases of AS, either the child does not get any copy, or the child receives two copies – each from mother and father. Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system.