Cancer & Disease Research
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
How one cell transplant platform is poised to tackle complex diseases
Lineage Cell Therapeutics CEO Brian Culley discusses the company's cell therapy technology with ScienceBoard.net. The technology involves the transplantation of differentiated stem cells as part of an off-the-shelf allogeneic cell therapy. Read More
New tool sheds light on transient protein interactions in cells
Researchers have developed a novel chemical tool for elucidating protein interaction networks within cells. The technique, details of which were published in Molecular Cell on April 23, allows for the visualization of protein-protein interactions. Read More
A CRISPR on-off switch for genes controls expression without altering DNA
A new gene silencing tool allows scientists to switch genes on and off without altering genetic sequences. The tool, described in a paper published in Cell on April 9, uses a modified CRISPR-Cas9 system to introduce reversible epigenetic changes that control gene expression. Read More
New gene therapy could be effective in treating complex polygenic conditions
Scientists are applying gene therapy approaches in a new way by simultaneously administering a combination of cargos to treat complex polygenic neurodegenerative diseases with no single genetic cause. Details of the combination gene therapy in two animal models were detailed in a March 31 Science Advances article. Read More
Gene therapy based on transcription factors could be effective against Alzheimer's
A type of gene therapy using transcription factors to target DNA has been shown to dramatically reduce levels of the harmful protein tau in preclinical mouse models of Alzheimer's disease. The findings, published in Science Advances on March 19, suggest that the therapy could easily translate to the clinic and be effective in humans. Read More
Customized gene therapies successfully target rare eye diseases
Can gene therapies prove effective for the treatment of rare genetic diseases? One company may have found a solution that incorporates a high degree of customization, manufacturing expertise, and years' worth of knowledge. Read More
Imaging finds tooth decay's root, may lead to ways to fight it
Atom probe tomography and other techniques revealed that human enamel contains small chemical flaws that may affect the resiliency of the fundamental building blocks of teeth, in a study published July 2 in Nature. Read More
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