22 Results for 'Gene Therapy'
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HarborSite: Precise and Efficient Gene Editing for Next-Generation Gene Therapies
The HarborSite next-generation gene therapy platform enables integration of therapeutic genes into genomic safe harbors using highly specific and efficient recombinases to enable more predictable, safe and durable gene therapies.
MRBL: Next-Generation Gene Therapy for Molecular Skin Rejuvenation
The next-generation gene therapy for molecular skin rejuvenation combines a comprehensive target gene prediction with a novel transdermal delivery approach for therapeutic adenovirus-associated viruses. The platform targets monogenic disease indications in the skin, and extends the same targets to the treatment of common skin aging conditions.
Synthetic AAV Capsids for Advanced Gene Therapy
The protein shell (capsid) of Adeno-associated viruses (AAV) are presently the most promising delivery vehicles for various in vivo gene therapies. AAVs are non-pathogenic and, through past engineering efforts, have become safe due to their inability to integrate into and damage the genome of target cells. Rather, the delivered DNA containing a therapeutic gene of...
Video/AnimationLight-Seq: Light-Directed In Situ Barcoding of BiomoleculesThis animation explains how the Light-Seq technology works to barcode and deep-sequence selected cell populations in tissue samples, and how the team applied it to the analysis of distinct and rare cells in the mouse retina. Credit: Wyss Institute at Harvard University.
Audio/PodcastIlluminating Biological Context with Josie Kishi – Translation by Fifty YearsTechnologies like next-generation sequencing allow us to understand which RNA transcripts and proteins are expressed in biological tissues. However, it’s often equally important to understand how cells or molecules are positioned relative to one another! Whether it be a cell changing its shape, an organelle ramping up a metabolic process, or a DNA molecule traveling...
Video/AnimationeToehold: an RNA-detecting control element for use in RNA therapeutics, diagnostics and cell therapiesThis animation shows an example of an eToehold that detects and signals the presence of a specific viral RNA in a human cell. After the virus has injected its RNA into a host cell, the RNA acts as a “trigger RNA” by binding to a complementary sequence within the eToehold specifically engineered for its detection....
Video/AnimationImproving Canine HealthspanA Wyss Institute technology that can treat multiple age-related diseases is now being developed by Rejuvenate Bio into a treatment for mitral valve disease and other deadly conditions in dogs, with the goal of helping man’s best friend live longer, healthier lives. Credit: Wyss Institute at Harvard University
Video/AnimationAAV Capsid EngineeringWyss researchers have created a high-throughput platform to generate an Adeno-associated virus 2 (AAV2) library containing 200,000 variants, each carrying a distinct mutation in the virus capsid protein. Their analysis identified capsid changes that enhanced “homing” potential to specific organs in mice and virus viability, as well as a new protein hidden in the capsid-encoding...