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Macrophage cell therapy: a new hope for chronic liver disease patients

Drug Target Review

Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years. Could you describe the platform of macrophage biology and cell engineering used by Resolution Therapeutics in developing their cell therapies?

Therapies 114
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Study reveals how cancer immunotherapy may cause heart inflammation in some patients

Broad Institute

We are incredibly grateful to each and every patient who partnered with us, all those involved in their clinical care, and the exceptional team in our lab who made this research possible.” “This work provides a biological foundation for testing more targeted therapies for myocarditis due to an immune checkpoint inhibitor.

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Revolutionary nanoparticles enable gene-editing in lungs

Drug Target Review

Scientists from the Massachusetts Institute of Technology (MIT) and the University of Massachusetts Medical School (UMass), US, have collaborated to create a novel type of nanoparticle that can deliver messenger RNA that encodes for beneficial proteins to the lungs. The study appears in Nature Biotechnology.

RNA 98
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To help his daughter living with an ultra-rare disorder, this dad brought together a squad of genetic detectives

Broad Institute

By Leah Eisenstadt, Broad Communications October 23, 2024 Credit: Courtesy of the Broadbent family Brian and Julia Broadbent are raising their daughters Claire, top left, and Emma, seated, who is the first person to be diagnosed with a rare genetic disorder caused by the long noncoding RNA CHASERR.

DNA 128
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A common fatty acid may help restore healthy vaginal bacteria after infection

Broad Institute

Using RNA sequencing and working with the Broad’s Metabolomics Platform and collaborators at St. I’m so proud of this team and the partnership we formed to follow the science and set up some major positive impacts on women’s health,” Blainey said.

Hospitals 121
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Could Gene Therapy Cure Sickle Cell Disease? Two New Studies Raise Hopes

The Pharma Data

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both of the new gene therapy studies were published online Dec. SATURDAY, Dec. 5 in the New England Journal of Medicine.

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SRP-001: redefining pain treatment with a safer, non-opioid analgesic

Drug Target Review

Using the 10X Genomics Chromium platform, we conducted ribonucleic acid (RNA) sequencing on the midbrain’s PAG region in animals treated with SRP-001, ApAP and a vehicle control, focusing on gene expression changes related to pain processing. was founded in 2016 to develop safer, non-opioid therapies for acute and chronic pain.