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Researchers speed identification of DNA regions that regulate gene expression

Scienmag

Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Credit: St. Jude Children’s Research Hospital St.

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Logistics in Focus: Orchestrating the Challenges of Cell Therapy

Worldwide Clinical Trials

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. The global nature of cell and gene therapy trials adds another layer of complexity.

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New method boosts the study of regulation of gene activity

Scienmag

One way cells can control the activities of their genes is by adding small chemical modifications to the DNA that determine which genes are turned on or off. Researchers have found that in bacteria DNA methylation plays a role in regulating virulence, reproduction and gene […].

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Gene therapy specialist bluebird exits “untenable” European market

pharmaphorum

Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. — Brad Loncar (@bradloncar) August 9, 2021.

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The structure of DNA is found to be actively involved in genome regulation

Scienmag

The two meters of -stretched- DNA contained in human cells are continuously twisting and untwisting to give access to genetic information: when a gene is expressed to generate a protein, the two strands of DNA are separated to give access to all the machinery necessary for this expression, resulting in an excessive accumulation of coiling […]. (..)

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BioMarin Gene Therapy Halted by FDA Over Safety Concerns

XTalks

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

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FDA slaps clinical hold on BioMarin’s PKU gene therapy

pharmaphorum

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. “We are committed to understand and mitigate any risk of cancer causation.”