Biotechnology - A New Cas9 mRNA-Based Therapy Holds Promise to Treat Muscular Dystrophy
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Smarter Decisions, Stronger Medicines: The Role of Advanced AnalyticsSmarter Decisions, Stronger Medicines: The Role of Advanced Analytics

Process variability is one of the biggest challenges in pharmaceutical scale-up and manufacturing. This eBook explores how greater process understanding, real-time analytical insight, and process monitoring strategies can help reduce risk, accelerate scale-up, and support consistent product quality. Discover approaches that help teams make more confident decisions from development through production.

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    A New Cas9 mRNA-Based Therapy Holds Promise to Treat Muscular DystrophyA New Cas9 mRNA-Based Therapy Holds Promise to Treat Muscular Dystrophy
 
Researchers report a promising Cas9 mRNA therapy for limb-girdle muscular dystrophy, a group of inherited disorders causing progressive shoulder and hip weakness. In a dystrophic mouse model with a pathogenic mutation, quantitative confocal imaging showed that Cas9 mRNA-based editing restored Telethonin muscle protein expression and correct localization at the sarcomeric Z-disc with Titin, indicating potential recovery of muscle function.
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    Discover Innovations Shaping Biopharmaceutical ManufacturingDiscover Innovations Shaping Biopharmaceutical Manufacturing
 
From biologics production to advanced bioprocessing technologies, pharmaceutical manufacturing continues to evolve alongside biotechnology innovation.

This Industry Focus eBook examines emerging manufacturing approaches, analytical advances, and production strategies, offering expert insights into the technologies helping translate scientific discoveries into scalable, high-quality therapeutic products.
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 Next-Generation CRISPR Tools Transform Gene Editing And Gene Therapy
 
Next-Generation CRISPR Tools Transform Gene Editing And Gene TherapyEmerging gene editing platforms are revolutionizing precision medicine, enabling safe and effective genetic modifications for diverse therapeutic applications.
 
 
 Cherry Bark Design Turns Cellulose Into Spider Silk-Like Tough Fibers
 
Cherry Bark Design Turns Cellulose Into Spider Silk-Like Tough FibersResearchers developed regenerated cellulose fibers inspired by cherry bark’s helical architecture, using microfluidic spinning to create a biaxially oriented structure with spider silk-like toughness. The fibers reached 553 MPa tensile strength, 41% fracture strain, and 184 MJ m?³ toughness, and could be woven into durable fabrics for future sustainable textiles and structural materials.
 
 
 Drug Boosts Axon Regrowth In Human Brain-Spinal Cord Model
 
Drug Boosts Axon Regrowth In Human Brain-Spinal Cord ModelThis study presents a human organoid model that identifies genetic factors limiting nerve regeneration and highlights lynestrenol's role in promoting repair.
 
 
 Building A Soft Robotic Heart That Mimics HFpEF Progression
 
Building A Soft Robotic Heart That Mimics HFpEF ProgressionResearchers developed a soft robotic heart model that replicates HFpEF, enhancing understanding of the disease and supporting innovative treatment strategies.
 
 
 Building More Realistic Human Tissue Models With Assembloids
 
Building More Realistic Human Tissue Models With AssembloidsAssembloids combine organoids and specialized cells, creating advanced models for drug testing and regenerative medicine with improved physiological relevance.
 
 
 Advancing Human Muscle Models for Drug Development and Preclinical Testing
 
Advancing Human Muscle Models for Drug Development and Preclinical TestingTransforming drug development, Myomaker Bio's lab-grown muscle platforms offer human-relevant models for effective preclinical testing and risk reduction.
 
 
 First-in-the-world gene therapy delivers missing gene directly to infant’s brain, marking historic milestone in precision medicine
 
First-in-the-world gene therapy delivers missing gene directly to infant’s brain, marking historic milestone in precision medicineAn eight-month-old baby from Israel has become the first human ever to receive an experimental gene therapy designed to replace a missing gene responsible for a devastating neurological disease that until now offered no hope of survival.
 
 
 New SMArT platform improves safety of CRISPR gene editing
 
New SMArT platform improves safety of CRISPR gene editingA team of researchers led by Luigi Naldini at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) has developed a new strategy to significantly improve the precision and safety of CRISPR-Cas9 gene editing in human blood stem cells, potentially overcoming one of the major barriers limiting broader clinical application of genome editing therapies.
 
 
 Biotechnology breakthrough could revolutionize treatment for neurodegenerative diseases
 
Biotechnology breakthrough could revolutionize treatment for neurodegenerative diseasesA research team from the School of Biomedical Sciences at the LKS Faculty of Medicine, the University of Hong Kong (HKUMed), has achieved a significant breakthrough in biotechnology that could revolutionize treatment strategies for neurodegenerative diseases.
 
 
 Synthetic biology platform unmasks hidden bacterial targets of bacteriophages
 
Synthetic biology platform unmasks hidden bacterial targets of bacteriophagesAn interdisciplinary team of Rice University researchers has uncovered previously unknown relationships between bacteriophages - viruses that infect bacteria - and their bacterial hosts, offering a powerful new tool for next-generation microbiome engineering.
 
 

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