Remove Gene Expression Remove Protein Remove Regulation
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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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EHA 2023: Kura Oncology’s menin inhibitor ziftomenib shows impressive Phase I results

Pharmaceutical Technology

Ziftomenib targets the menin-KMT2a protein-protein interaction that may occur in patients with KMT2ar or NPM1m genetic alterations. The aberrant protein complex is essential for regulating leukaemia-promoting gene expression.

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

XTalks

The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (or miRNA) and its role in post-transcriptional gene regulation. However, microRNAs can bind to this mRNA, preventing it from functioning.

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AI-designed protein awakens silenced genes, one by one

The Pharma Data

By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. The chemical modifications that regulate gene activity are called epigenetic markers. it can be reawakened.

Protein 52
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Leading innovators in transcription factors for genetically modified cells for the pharmaceutical industry

Pharmaceutical Technology

Innovation S-curve for the pharmaceutical industry Transcription factors for genetically modified cells is a key innovation area in the pharmaceutical industry Transcription factors are proteins which control gene expression by controlling the rate of transcription of genetic information from DNA to RNA.

Genetics 130
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Gene editing: beyond the hype

pharmaphorum

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

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AZ, Scorpion strike deal on elusive transcription factor targets in cancer

pharmaphorum

AstraZeneca has expanded its efforts to develop new therapies targeting transcription factors – proteins that regulate processes in the cell that have become a hot topic for drug discovery in cancer and other diseases.

Protein 59