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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.

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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.

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Targeting the immunotherapy potential of cytokines IL-12 and IL-18 with new advancements in protein engineering

Drug Target Review

We are in an era of immuno-oncology (IO) revolution with many approved therapies now available to treat a broad range of cancers. For the last several years, the field has worked to unlock the potential of IO therapies for additional tumour types, and has explored options beyond checkpoint inhibitors.

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New image-based cellular profiling tool peers deeply into metabolic biology

Broad Institute

Typically, studying drug therapies is an expensive and time-consuming undertaking. That teaches us far more than looking for just a single informative data point.” The researchers also looked at the tool’s ability to detect changes in cellular phenotypes after exposure to drugs and small molecule compounds.

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Women in STEM with Charlotte Owens

Drug Target Review

After medical school and residency I decided to dedicate my life to women and specialised in obstetrics and gynaecology. I graduated with my BS in physiology and later attended the University of Michigan Medical School. Biotechnology revolutionises healthcare : Biotechnology is transforming medicine and healthcare.

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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.

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CRISPR Cas-9 System Doubles Life Expectancy in Mice with Aggressive Cancers

The Pharma Data

Back in September, Vertex Pharmaceuticals and CRISPR Therapeutics – the company behind the CRISPR Cas-9 platform – announced that the European Medicines Agency (EMA) had granted Priority Medicines (PRIME) designation to CTX001, an investigational ex vivo CRISPR Cas-9 gene-edited therapy for the treatment of severe sickle cell disease.

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