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Antisense therapy restores fragile X protein production in human cells

Science Daily: Pharmacology News

An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome.

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An immune factor in the brain plays critical roles in neuron function and aging

Broad Institute

A new study reveals that C1q can also enter neurons, where it influences protein production, and can accumulate within neurons over time. They also suggest that C1q influences learning, memory, and flexibility in the adult brain, potentially by interacting with specific complexes within neurons that impact protein production.

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Finding the right CDMO partner for cell line development

Drug Target Review

Together, these attributes provide a strong foundation for protein expression with enough adaptability to produce much of the commercial and therapeutic protein market. Skilled CDMOs can also locate or create favourable genomic microenvironments where transcription factors are efficiently recruited, further boosting protein production.

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Messenger RNAs with multiple “tails” could lead to more effective therapeutics

Broad Institute

Messenger RNAs with multiple “tails” could lead to more effective therapeutics By Corie Lok March 22, 2024 Breadcrumb Home Messenger RNAs with multiple “tails” could lead to more effective therapeutics Scientists have engineered long lasting mRNAs that increased therapeutic protein production in cells and animals.

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Merkin Prize in Biomedical Technology awarded to F. William Studier for development of widely used protein- and RNA-production platform

Broad Institute

William Studier for development of widely used protein- and RNA-production platform By Corie Lok May 14, 2024 Breadcrumb Home Merkin Prize in Biomedical Technology awarded to F. Merkin Prize in Biomedical Technology awarded to F.

RNA 84
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Structural biology: Molecular scissors caught in the act

SCIENMAG: Medicine & Health

Transfer RNAs (tRNAs) are among the most common types of RNA in a cell and are indispensable for protein production in all known organisms.

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Sarepta data show new Duchenne drug’s potency, but highlight side effects

BioPharma Drive: Drug Pricing

A successor medicine to Sarepta's Exondys 51 appeared better at boosting dystrophin protein production, but was associated with electrolyte imbalances.