Broad Institute

JANUARY 8, 2024

Now the researchers describe how they re-engineered both eVLPs and parts of the prime editing protein and RNA machinery to boost editing efficiency up to 170 times in human cells compared to the previous eVLPs that deliver base editors. By Sarah C.P.

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Drug Target Review

DECEMBER 6, 2023

Dear readers, RNA, or ribonucleic acid, plays a pivotal role in the intricate dance of cellular processes. We are committed to providing you with the highest quality content, and we believe that this report will deliver a different perspective on the topics covered.

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RNA DNA Vaccine Drug Development 52

BioPharma Drive: Drug Pricing

JUNE 3, 2024

With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.

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Science RNA Therapies Disease 57

Broad Institute

NOVEMBER 23, 2023

The work appears today in Science. The scientists found a surprising number and diversity of CRISPR systems, including ones that could make edits to DNA in human cells, others that can target RNA, and many with a variety of other functions. They studied several of the new systems in greater detail in the lab.

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The Pharma Data

JANUARY 5, 2021

Durham, North Carolina-based Ribometrix announced a strategic collaboration deal with Genentech , a Roche company, to identify and advance novel RNA-targeted small molecule therapeutics. Targeting RNA is believed to be a way to develop therapeutics for so-called undruggable proteins. The first identifies the 3D RNA motifs.

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RNA Small Molecule DNA Licensing 52

Broad Institute

OCTOBER 23, 2024

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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DNA RNA Disease Hospitals 128

Broad Institute

MAY 16, 2024

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor.

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Therapies FDA Approval Engineering Disease 137

Broad Institute

MARCH 22, 2024

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. and Virginia W.

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PLOS: DNA Science

MAY 16, 2024

These factors are converging to enable both identification of novel infectious diseases as well as microbial resistance, before these threats can impact public health, write a team from the European Society for Clinical Microbiology and Infectious Diseases in Frontiers in Science. COVID clearly caught us off guard.

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Codon

JANUARY 24, 2024

This is especially true in medicine, where the temporal gap between the initiation of the disease (a random genetic mutation, say) and the appearance of symptoms makes finding the root cause difficult. A capillary shrinks over time – it’s a temporal process – but science is often limited to static snapshots.

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Science Disease RNA Research 70

Drug Target Review

APRIL 26, 2023

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.

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RNA Medical Schools Engineering DNA 98

Broad Institute

JUNE 29, 2023

The method, described today in Science , provides a way for scientists to learn new details on how translation is regulated within individual cell types and how that changes in disease. By Sarah C.P. Williams June 29, 2023 Credit: Zeng H, Huang J, Ren J, et al. Paper cited Zeng, H. Online June 29, 2023. DOI: 10.1126/science.add3067

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KIF1A

MAY 4, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. By talking to groups with these mutations, we may identify other diseases whose patients have KIF1A variants. Biotechnology program at Columbia University.

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Drug Target Review

JULY 24, 2023

The results were published in Proceedings of National Academy of Sciences of United States of America (PNAS). Through phylogenetic analysis and microscopy techniques, they identified a nuclear-encoded apicoplast RNA polymerase σ subunit called ApSigma. The first identification was based on a bioinformatic analysis.

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RNA Regulations Bioinformatics Laboratories 98

PLOS: DNA Science

JULY 27, 2023

That information led, thanks to vaccine shelved from the first SARS circa 2003, to the rapid development and deployment of mRNA vaccines against the new infectious disease. Seventeen of the entire group (10.7%) had “unanticipated monogenic disease risks (uMDRs).”

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Broad Institute

AUGUST 31, 2023

Related News Next generation prime editing systems move closer to possible therapeutic applications New CRISPR genome editing system offers a wide range of versatility in human cells Prime editing technologies allow scientists to precisely edit the genome in a variety of ways and could one day be used to treat genetic diseases.

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Broad Institute

JUNE 28, 2023

Today, scientists use these reactions to produce customizable DNA and RNA molecules that enable genetic sequencing, drug and vaccine development, pathogen tests, cancer diagnostics, and many aspects of basic biomedical research. “I Longer synthetic DNA and RNA molecules are also critical for modern biologic drugs.

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Drug Target Review

NOVEMBER 9, 2023

A team of neurobiologists at The Molecular Neuroscience Unit at the Okinawa Institute of Science and Technology (OIST) has made a significant breakthrough by connecting sensory neurons’ survival and pathology with the way messenger RNAs (mRNAs) are transported inside these cells. References 1 Alcaraz G, Campanac E, Carlier E, et al.

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RNA Disease Science Research 98

Broad Institute

JANUARY 30, 2024

More bottles on the medicine shelf would help, but Bhattacharyya, who’s now also a research scientist, realized that science might lead to better tools for diagnosing and treating such challenging cases. FROM STARS TO CELLS Bhattacharyya’s interest in science began not in the lab, but in the sky.

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Hospitals Medical Schools Virus Science 137

Broad Institute

AUGUST 19, 2024

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. Using RNA sequencing and working with the Broad’s Metabolomics Platform and collaborators at St. Paper cited Zhu M et al.

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Hospitals Treatment Medical Schools Science 121

Broad Institute

AUGUST 31, 2023

We were surprised to see that just missing one copy of this gene can induce many changes at the RNA level, at the protein level, at the functional level, and at the behavioral level,” said Farsi. Tags: Stanley Center for Psychiatric Research Psychiatric Disease Paper(s) cited: Farsi Z, et al. Aug 31, 2023. DOI: 10.1016/j.neuron.2023.08.004.

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KIF1A

JANUARY 20, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Instead, this is what is called a splicing mutation; it occurs at the boundary between coding DNA that is read into RNA and protein, and noncoding DNA that is excluded.

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Drug Target Review

SEPTEMBER 4, 2023

There have been several studies that have reported the ability of infectious disease agents to be drug resistant. Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. They could be spread by physical contact, airborne, waterborne, foodborne, or vector-borne.

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Drug Target Review

JULY 23, 2024

I studied Natural Sciences at the University of Cambridge, where I was exposed to a range of disciplines from chemistry to physiology, specialising in my last year in biochemistry. Shortly after finishing my studies, I landed my first job in industry working on cell biology research for several disease indications.

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Small Molecule Targeted Protein Degradation Science Disease 115

Broad Institute

MARCH 8, 2024

By Claire Hendershot March 8, 2024 Credit: National Institute of Allergy and Infectious Diseases, National Institutes of Health via U.S. Related news #WhyIScience Q&A: How a global collaboration is boosting science and public health in Senegal Why did you and your colleagues in Senegal decide to do this study?

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Research Disease Medical Schools RNA 111

Broad Institute

OCTOBER 28, 2024

To speed up the annotation step, the Broad Institute’s Data Sciences Platform (DSP) has developed a new search engine that automates much of this process by using machine learning to search data on more than 50 million annotated single cells. Tags: Data Sciences Platform Machine Learning Single Cell Is CAS now available to use?

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Engineering Cell Biology Disease RNA 119

PLOS: DNA Science

JULY 13, 2023

Most studies on senescence are related to chronic inflammation, cancer, and age-related diseases. Analyzing RNA Revealed the Regeneration The researchers analyzed RNA to reconstruct the steps to regeneration. The researchers discovered RNA transcripts known to be involved in senescence in the disembodied heads.

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RNA DNA Research Disease 97

KIF1A

JULY 29, 2023

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. KIF1A in New York: The 2023 KAND Family & Scientific Engagement Conference We won’t be posting next Saturday, but not for a lack of science!

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The Pharma Data

SEPTEMBER 28, 2020

This included compiling RNA data, as well as other data points such as patient demographics, clinical treatment arm and patient diagnosis. For years, patients diagnosed with a disease often received the same treatment. And for some people, that treatment worked.

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Science Clinical Trials RNA Treatment 52

Drug Target Review

AUGUST 9, 2023

Published on bit.bio’s website, this article authored by Dr Tony Oosterveen dives into the close relationship between FTD and ALS with a focus on mechanisms underlying the neuropathology of both diseases. ALS is the most common motor neuron disease and is estimated to affect more than 80,000 people worldwide.

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RNA Regulations Disease DNA 98

Broad Institute

MAY 2, 2024

But as an undergraduate at MIT, Martin found she could study biology and computer science and still row. In 2018, she graduated with a bachelor’s degree in computer science and biology and joined the lab of Gad Getz at the Broad Institute of MIT and Harvard to complete her master’s. The act of discovery always appealed to me.

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Codon

DECEMBER 12, 2024

” The report accompanies an article in Science , also released today, entitled “Confronting Risks of Mirror Life.” For the article in Science , more than 30 scientists from ten countries are calling on the broader community to confront the risks of mirrored life. ” But what, exactly, is a mirrored organism?

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The Pharma Data

MARCH 9, 2022

Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets. About Novartis.

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Drug Target Review

FEBRUARY 6, 2024

Prostate cancers detected at the local stages, when the disease is limited to the prostate and its immediately surrounding regions, have a nearly 100 percent five-year survival rate. Advances in DNA and RNA-focused molecular diagnostic methods have made blood-based multi-cancer early detection (MCED) tests a reality, but not for all patients.

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Broad Institute

DECEMBER 13, 2023

Related news Researchers map brain cell changes in Alzheimer’s disease Courtesy of the Chen and Macosko labs. The researchers also discovered clues about cellular function and the potential roles of brain structures in disease. Courtesy of the Chen and Macosko labs. Courtesy of the Chen and Macosko labs.

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Science Daily: Pharmacology News

JULY 27, 2023

In a step forward in the development of genetic medicines, researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

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RNA Research Therapies Disease 57

Dark Matter Blog

JUNE 21, 2023

Since our company’s founding in 2015, we have taken the long view and been singularly focused on building an extremely flexible and broadly applicable platform that can develop a host of RNA‑targeted small molecules (rSMs) to deliver precision medicines for dozens of targets that have been out of reach for conventional approaches.

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