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Slowing intracellular transport boosts potential for RNA therapies
Researchers at the University of Basel and Roche have discovered that slowing the intracellular transport of RNA-based drugs significantly improves their therapeutic impact.
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Duchenne muscular dystrophy
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New insights into the role of viral capsids in gene therapy safety
Viral capsid assembly and quality are key factors in gene therapy success and safety. Here, Dr Chelsea Pratt explores how cutting-edge tools are enabling scientists to tackle these challenges and improve patient outcomes.
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Mouse study shows lab-grown, self-sustainable muscle cells could repair diseases such as muscular dystrophy
A new study highlights how scientists have successfully cultivated human muscle stem cells capable of renewing themselves and repairing muscle tissue damage in mice, findings that could lead to therapeutics to treat muscular dystrophy disorder.
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Novel nanoparticles improve CRISPR gene editing
A study has used new synthetic lipids to deliver CRISPR gene editing tools into cells with up to 90 percent efficiency.
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CRISPR-Chip enables digital detection of DNA without amplification
Researchers have found multiple applications for the CRISPR (Clustered Regularly Interspersed Short Palindromic Repeats) gene editing technology since it came into use by the scientific community...
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A step closer to preventing autoimmune response to gene therapy
Scientists in the U.S. have demonstrated an effective means of administering gene therapy in mice, without the dangerous autoimmune reaction that often occurs.
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CRISPR engineered to edit single RNA letters
New 'REPAIR' system edits RNA, rather than DNA, without permanently affecting the genome...
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Macrophages play critical role in sustained delivery of MD therapy
Inflammatory cells retain drug after it disappears from circulation, improving delivery to regenerating muscle cells...
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Promising therapeutic approach for improving mitochondrial function
Scientists have made progress in the development of products that improve mitochondrial function...
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Screening for genetic diseases & chromosomal defects with a single biopsy
Couples who are undergoing pre-implantation genetic diagnosis (PGD) in order to avoid transmission of inherited diseases, such as Duchenne muscular dystrophy or cystic fibrosis, should also have their embryos screened for abnormal numbers of chromosomes at the same time, say Italian researchers.
