Scientists release open-source platform for large-scale biological simulations
A team of scientists has released BioDynaMo, an open-source simulation platform for the life sciences. The new computational tool can perform complex simulations of biological processes in fields such as neuroscience, oncology, and epidemiology. The software was detailed in a paper published on September 16 in Bioinformatics. Read More
New gene therapy platform shows potential against certain muscle diseases
Scientists have engineered a new family of adeno-associated viruses (AAVs) to deliver therapeutic genes to muscle tissue at 100 to 250 times lower doses than previous AAV vectors, potentially reducing the risk of liver damage and other serious side effects, according to a new study published in Cell on September 9. Read More
Antisense oligonucleotide therapies get delivered across the blood-brain barrier
Researchers have demonstrated that heteroduplex oligonucleotide drugs conjugated with cholesterol efficiently cross the blood-brain barrier and can be used to treat central nervous system diseases. The findings were published in Nature Biotechnology on August 12. Read More
Optogenetic gene therapy provides hope for retinitis pigmentosa patients
Optogenetic therapy bypasses traditional methods of repairing defective photoreceptors in the eye by targeting other retinal cells. Recently, three examples of optogenetic therapeutics under development highlight many of the challenges this type of gene therapy faces for the treatment of a devastating genetic eye disease. Read More
Gene therapy tackles 2 rare childhood diseases 2 different ways
Two new studies point to the efficient use of gene therapy to treat deadly childhood genetic diseases. The first study used direct gene therapy infusion to treat a rare neurodevelopmental disorder, while the second study used prenatal base editing in mouse models as proof of concept for the treatment of genetic diseases. These vastly different approaches show the promise of gene therapy for a variety of devastating diseases. Read More
How spatial biology can impact clinical translational research
Building on the success of single-cell technologies, 10X Genomics' Michael Schnall-Levin, PhD, discusses with ScienceBoard.net how the company entered the spatial biology field and how it continues to support the advancement of various clinical translational research efforts. Read More
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