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Home » Broad Institute

Articles Tagged with ''Broad Institute''

DNA double helix under a magnifying glass
Drug design, drug delivery & technologies

Base and prime editions to achieve genetic cure

May 21, 2025
By Mar de Miguel
No Comments
Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base editing and one clinical assay for prime editing. The 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) marked a historic milestone this year by presenting the first case of treatment with base editors of a baby with a deadly metabolic disease.
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Antibodies
Immuno-oncology

A single antibody to attack two epitopes for cholangiocarcinoma driven by FGFR2 fusions

April 30, 2025
Abnormal fusion of the FGFR2 gene, encoding the fibroblast growth factor receptor 2, occurs in several types of cancer, including in up to 15% of cases of intrahepatic cholangiocarcinoma.
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Neurology/psychiatric

Broad Institute advances gene therapy for prion disease

March 3, 2025
The new gene therapy aims to address the root cause of prion disease by using the CHARM epigenetic editing platform from the Whitehead Institute to target and silence the gene that codes for the disease-causing protein.
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Histological staining of the caudate nucleus from a person with Huntington’s disease.
Neurology/psychiatric

Huntington’s neurodegeneration starts at 150 repeat expansions

Jan. 17, 2025
By Mar de Miguel
Having 35 copies of the CAG triplet in the gene that causes Huntington’s disease is not a problem. Inheriting 40 could be a sign that goes unnoticed for decades, until reaching 80. From there, the process accelerates and neural death occurs when reaching 150 repeats. Huntington’s disease neurodegeneration is not determined by what, but by how much, according to a study conducted at the Broad Institute.
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Lab mouse
Genetic/congenital

Inhibiting GSK-3α/β rescues mice from CTNNB1 syndrome

Sep. 2, 2024
CTNNB1 syndrome is a neurodevelopmental disorder characterized by intellectual disability, global developmental delay, microcephaly and motor disabilities, among others, caused by pathogenic loss-of-function variants in the CTNNB1 gene, which encodes β-catenin. This syndrome has no treatment option, with only supportive care available. To address this unmet medical need, researchers from the Broad Institute and Tufts University School of Biomedical Sciences have developed a Ctnnb1 germline heterozygote murine model that mimics the human CTNNB1 syndrome.
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Graphic showing particles delivering cargo to a mouse brain
Drug Design, Drug Delivery & Technologies

Better gene editing technology partially restores vision in mice

Jan. 25, 2024
Researchers from Broad Institute and Harvard University presented the discovery of all-in-one virus-like particles (VLPs) designed to deliver prime editor (PE) ribonucleoprotein (RNP) complexes into mammalian cells.
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Illustration of DNA, digestive system
Biomarkers

Large study reveals protective effect of CFTR variants against inflammatory bowel disease

Nov. 6, 2023
It is well known that mutations in the cystic fibrosis transmembrane regulator (CFTR) gene are causative of cystic fibrosis, a lethal autosomal recessive Mendelian disorder. Several studies have also pointed to an association between CFTR mutations and inflammatory bowel disease (IBD).
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Cancer

Bayer and Broad Institute extend longstanding cancer research collaboration

Nov. 3, 2023
Bayer AG and the Broad Institute of MIT and Harvard have extended their research collaboration by an additional 5 years.
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Immuno-oncology

Abbvie and Calico Life Sciences publish data illustrating preclinical antitumor efficacy of ABBV-CLS-484

Oct. 6, 2023
Current therapies based on immune checkpoint blockade are effective and offer a valid option for treatment, but many patients develop either primary or acquired resistance to treatment. Previous research has shown that the deletion of protein tyrosine phosphatases PTPN2 and PTPN1 results in an increase in the sensitization of tumor cells and the promotion of antitumor immunity.
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Endocrine/Metabolic

Broad Institute and Novo Nordisk collaborate in type 2 diabetes and cardiac fibrosis

Sep. 7, 2023
The Broad Institute of MIT and Harvard has announced a new research alliance with Novo Nordisk A/S in diabetes and cardiometabolic diseases. The collaboration will focus on advancing three programs over the next 3 years.
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