Antisense therapy restores fragile X protein production in human cells
Science Daily: Pharmacology News
JULY 5, 2023
An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome.
Science Daily: Pharmacology News
JULY 5, 2023
An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome.
The Pharma Data
DECEMBER 3, 2020
Highlights from the Company’s presentations include: BUTTERFLY Observational Study – Baseline Analysis. A review of the study design for MONARCH, the Company’s ongoing Phase 1/2a study of STK-001 in children and adolescents with Dravet syndrome will be presented during a poster session by Linda Laux, M.D., Antiepileptic Drugs / 7B.
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Drug Target Review
MARCH 15, 2024
Transposons preferentially integrate the gene of interest into active transcriptional sites, facilitating rapid cell line development and higher titers of the protein drug. Engineering of Chinese hamster ovary cell lipid metabolism results in an expanded ER and enhanced recombinant biotherapeutic protein production.
The Pharma Data
JANUARY 16, 2021
Etta Biotech”), to set up a high titer transient protein expression platform for high quality protein production using JS Bio’s transient transfection media. JS Bio becomes the exclusive cell culture supplier for Etta Biotech’s transient transfection high titer protein expression platform.
The Pharma Data
OCTOBER 18, 2021
Tofersen binds to SOD1 mRNA, allowing for its declination by RNase-H in an trouble to reduce conflation of SOD1 protein product. About Tofersen Tofersen is an antisense medicine being estimated for the implicit treatment of SOD1-ALS.
The Pharma Data
OCTOBER 14, 2020
Live Webcast : accessible from the Company’s website at www.eloxxpharma.com under Events and Presentations or with this link: [link]. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. International Dial-in Number: (210) 874-7715. Conference ID : 9467336. About Eloxx Pharmaceuticals.
The Pharma Data
MARCH 23, 2022
Early in infection, it appears that host cellular proteins are in defense mode against Ebola virus polymerase and the viral genetic material. “This presents additional hurdles for polymerase,” says Fang. “The virus hijacks this protein and makes it do something different than its normal job in cells.
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