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Scientists create next generation of tools in battle against brain disease

Broad Institute

Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders. The tools are known as enhancer AAV vectors.

Disease 72
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Identification of Covalent Cyclic Peptide Inhibitors Targeting Protein–Protein Interactions Using Phage Display

Covalent Modifiers

4c15843 Peptide macrocycles are promising therapeutics for a variety of disease indications due to their overall metabolic stability and potential to make highly selective binding interactions with targets. Boyd, Lei Wang, Ralf Bartenschlager, and Matthew Bogyo Journal of the American Chemical Society 2025 DOI: 10.1021/jacs.4c15843

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Exploration of the Active Components and Mechanism of Jiegeng (Platycodonis Radix) in the Treatment of Influenza Virus Pneumonia Through Network Pharmacology Analysis and Experimental Verification

Chemical Biology and Drug Design

Combined with network pharmacology and experimental verification, this study found that platycodin D and luteolin in Jiegeng ( Platycodonis Radix ) may regulate inflammation through the IL-17 signaling pathway to treat influenza virus pneumonia.

Virus 100
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The future of CNS drug development: signs of real progress

Drug Target Review

Disorders such as Alzheimer’s disease , primary glioblastoma and amyotrophic lateral sclerosis (ALS) all affect the CNS and are considered fatal, while more common conditions, including depression , strokes and epilepsy , require long-term treatments. Viral vectors use a virus’s blueprint to deliver genetic material into cells.

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Prime editing treats childhood brain disease in mice

Broad Institute

There is no cure or effective treatment for this rare genetic disease, but new research suggests a potential path to one.& & The new study, published in Cell , is the first time prime editing has been used to treat a neurological disease in animals, offering hope for treating people with AHC and other genetic brain disorders.

Disease 141
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Bio-Techne Joins Forces with USP to Speed Up Development of mAbs and Gene Therapies

The Pharma Data

Biologic drugs, including monoclonal antibodies, have become essential in the treatment of a wide array of conditions, from autoimmune diseases to various cancers. To date, more than 160 monoclonal antibody therapies targeting nearly 100 disease-related proteins have received regulatory approval globally.

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Gene editing extends lifespan in mouse model of prion disease

Broad Institute

By Allessandra DiCorato January 14, 2025 Credit: Maria Nemchuk, Broad Communications Eric Minikel and Sonia Vallabh run a lab with a singular focus: preventing and treating prion disease within their lifetime. Its a huge privilege to have the opportunity to point these powerful new tools at our disease.

Disease 144