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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. Genetic literacy among clinicians may not be as high as you think.

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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.

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Delivering on the promise of gene editing

Drug Discovery World

These platforms all induce double strand cuts in the chromosomal DNA that can be sealed by the cell, leading to a specific gene disruption, or resulting in a new target site for inserting DNA segments. Gene editing tools can sometimes bind to unintended sites, typically because of sequence homologies and/or mismatch tolerance.

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CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. Better, faster, stronger: targeting cancer with precision by CRISPR-enhanced CAR T therapies Cancer is a global public health crisis that demands powerful, innovative solutions in the battle against it.

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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. GeneTx and Ultragenyx are co-developing the therapy.

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The use of base editing in stem-cell based therapies

Drug Discovery World

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.