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By Greta Friar, Whitehead Institute June 27, 2024 Images of a mouse brain show the effect of a technology called CHARM in turning off the expression of a gene in the brain. CHARMs are an elegant solution to the problem of silencing disease genes, and they have the potential to have an important position in the future of genetic medicines.” “The
There have been several studies that have reported the ability of infectious disease agents to be drug resistant. Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. They could be spread by physical contact, airborne, waterborne, foodborne, or vector-borne.
Heterogeneity in Hematological Cancers Due to their disease heterogeneity, hematological cancers are well-suited for personalized approaches. Molecular Biomarkers : Encompass various molecules, such as RNA and metabolites, to reflect the physiological state of the cells and their disease pathways.
This is a game-changer, especially in the fight against cancer and other complex diseases. These multifunctional small molecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. This can potentially correct the root causes of some diseases at the genetic level.
Some of the funds will be used for the development of therapeutics for oncology and other serious diseases through its proprietary ImmunoTAC technology platform. The ImmunoTAC platform pairs proprietary payloads that modulate key disease modifying pathways with monoclonal antibodies directed at specific disease sites.
The CureVac-GSK COVID-19 collaboration announced in February 2021 extends the existing strategic mRNA technology partnership both companies started in July 2020, which focuses on the development of new products based on CureVac’s second-generation RNA-technology for different targets in the field of infectious diseases.
Today, scientists use such reactions to produce the customizable DNA and RNA molecules that enable genetic sequencing, drug and vaccine development, pathogen tests, cancer diagnostics, and more. Wang concluded her presentation by expressing gratitude to Merkin. I’m really thankful for the support of the Merkin Fellowship,” she said. “It
Small snippets of double-stranded RNA were sprayed onto hot pepper plants to control a pest, called Frankliniella occidentalis. The RNA strands shut down a specific gene in the insects, and killed half after 7 days. RNA may offer a safer form of pest control in the future. The new sequences might help de-extinction efforts.
Gene Therapy Gene therapy is currently considered for diseases, often rare, that have no other cures. We have conducted over 130 in-life studies in the last five years (over 95% conducted in NHPs), including IND-enabling studies for rare diseases and CRISPR gene editing therapeutics in NHPs.
Forge Therapeutics received FDA clearance to initiate a Phase I/II trial of its FBX-101 for patients with Krabbe disease. Krabbe disease is a rare and fatal pediatric leukodystrophy caused by mutations in the galactosylceramidase (GALC) gene. ENPP1 deficiency is a rare, chronic, systemic, and progressive disease.
What are the key findings of Circio’s in vivo proof-of-concept for its circVec circular RNA platform technology compared to conventional mRNA-based expression with DNA vectors? Circular RNA (circRNA) has two major advantages versus mRNA in a vector-expression context. DNA vectors in mouse models?
The peak number of trials in the last five years and the regulatory approvals of three additional disease-modifying treatments for ALS in the previous eight years signal cause for hope that, as a community, we have the potential to transform this progressive and ultimately terminal neurodegenerative disorder into a treatable condition.
All patients deserve a gene therapy designed to address the genetic root cause of their disease with a single dose. The company’s commitment to SMA extends beyond gene therapy to branaplam (LMI070), an oral, once-weekly RNA splicing modulator also currently under development, to expand the treatment options for SMA patients. .
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