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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

One company leading the charge in this revolution is Alltrna , whose pioneering work in engineered transfer RNA (tRNA) therapeutics is offering new hope for patients with rare genetic diseases. These codons signal the premature end of protein translation, resulting in truncated or non-functional proteins.

Disease 80
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Key considerations before commencing cell line development

Drug Target Review

After binding, the transposase enzyme will excise the flanked section of DNA (termed the transposon) and insert it into sites in the genome at specific target sequences. A potential future for host cell lines There have been many recent advances in cell line engineering technologies and our understanding of CHO cells. Biotechnol.

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Gene editing extends lifespan in mouse model of prion disease

Broad Institute

The treatment, which uses base editing to make a single-letter change in DNA, reduced levels of the disease-causing prion protein in the brain by as much as 60 percent. They then administered the AAVs to mice infected with the human prion protein. But the researchers needed to deliver the base editors to the brain.

Disease 144
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Article FDA Thank You Draft guidance on potency assays for CGT products garners extensive stakeholder input

Agency IQ

Cell-based assays , while more complex to develop and use, offer another option for characterizing product activity at a slightly higher level, allowing sponsors to measure the further downstream effects of products (e.g., morphological cell changes or alterations in protein production or expression).

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Cave Coronavirus in Wuhan Lab Seeded COVID – The Truth Has Always Been Out There, in the Genetics

PLOS: DNA Science

Experiments at the Wuhan facility were done at the lowest two of the four standard biosafety levels, which were established at the dawn of recombinant DNA research in the 1970s. And a change to one specific part of the virus enabled it to locate and glom onto our cells, then slip inside and take over the protein production line.

Virus 52
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Codon Digest: Bacteria Resist Every Virus

Codon

🧪 Papers AI + Bio Protein-specific signal peptides for mammalian vector engineering. Evolutionary-scale prediction of atomic-level protein structure with a language model. Biological Engineering Generation of functional oocytes from male mice in vitro. Metabolic Engineering. Metabolic Engineering.

Virus 52
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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.