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Alnylam identifies gene to lower abdominal fat and decrease cardiovascular risk

Pharmaceutical Technology

Alnylam Pharmaceuticals and collaborators have identified rare mutations in the INHBE gene that is expressed in the liver, related to a lower waist-to-hip ratio for body mass index (BMI), which is often used as an indicator of abdominal fat and is correlated with the risk of type 2 diabetes (T2D) and coronary heart disease.

Gene 130
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Biologics – The Next Step in Revolutionary Medication

Roots Analysis

Biologics activate certain proteins or cells in your immune system to create specific responses to targets, while many conventional systemic drugs activate the entire immune system in a more generalized manner. This can be attributed to the fact that the production of biopharmaceuticals requires living expression systems.

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Cell-penetrating peptides as a delivery system for oligonucleotides

Drug Discovery World

Over the years, a range of different CPPs have been developed, including natural translocating proteins through to newly-designed computer-prediction sequences 3. Plus, in numerous in vitro experiments, the conjugate has been able to specifically enter ? How are cell-penetrating peptides used? Nanoparticles 6.

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What the Glycome Can Tell Us About Persistent HIV Infection

XTalks

Studies have identified the presence of two types of HIV-infected CD4+ T cells: ‘Transcriptionally inactive’ cells that do not typically produce viral RNA or viral proteins. This may help explain why HIV is adequately controlled, but not eradicated, with current treatments. The research study was published in Cell Reports.

RNA 52
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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

Angelman syndrome is a result of the missing or dysfunctional gene known as UBE3A. The gene is responsible for the normal and healthy development of an individual physically as well intellectually. Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system.

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RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. ASOs are short single-stranded nucleotides that bind to specific messenger RNAs and prevent the production of a particular protein.

RNA 40
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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.