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US FDA clears Transcenta’s IND for Rett syndrome treatment

Pharmaceutical Technology

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

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Biotech founder arrested in US amid murder-for-hire investigation

pharmaphorum

Gümrükcü is a specialist in gene expression profiling in stem cells, and has been working on ways to deliver stem cell transplants to treat cancer without the need for high-dose induction chemotherapy to destroy the patient’s bone marrow. .” ” If convicted, they face mandatory life in prison or the death penalty.

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Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

These modifications regulate gene expression without changing the sequence or structure of DNA. We can do this for multiple genes at the same time without any DNA damage, and in a way that can be reversed. It’s a great tool for controlling gene expression.”. pyogenes dCas9.

DNA 98
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Discovery of MicroRNA Genetic Regulation Leads American Scientists to Win 2024 Nobel Prize

XTalks

Their breakthrough revealed a previously unknown mechanism by which genes are controlled that is essential for the development and function of multicellular organisms, including humans. A gene contains instructions within our DNA.

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Nucleome raises £37.5m to shine light on ‘dark genome’

pharmaphorum

The dark genome – a loose term that covers non-coding regions of the genome that are capable of regulating the expression of genes, previously rather inaccurately referred to as junk DNA – is increasingly being explored by biopharma companies for new drug targets.

Genome 59
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Symvivo’s Oral COVID-19 Vaccine Enters Clinical Trials

XTalks

DNA vaccines designed using the platform consist of a combination of an advanced gene expression plasmid (pDNA) with gene(s) of interest, and a probiotic bacterium to deliver the vaccine directly to the gut, where it binds to intestinal epithelial cells.

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

Roots Analysis

Further, the transcription factors contain two molecular domains, the DNA binding domain and the activation domain. Moreover, some transcription factors contain additional domains, such as ligand binding domain in order to interact with chemical signals.

Gene 40