Embryonic Motor Neuron Programming Factors Reactivate Immature Gene Expression and Suppress ALS Pathologies in Postnatal Motor Neurons

Medicine, Basic Medical Sciences, Neurology, Life Sciences, Cell Biology, Biochemistry, Bioengineering, Genetics,
motor neuron   ALS   transcription factors   gene expression   neurodegeneration   reprogramming   gene therapy  

1. Academic Background and Research Motivation Degenerative diseases of motor neurons, such as Amyotrophic Lateral Sclerosis (ALS), have long been a key research area in neuroscience. ALS is characterized by adult onset, with progressive degeneration of motor neurons leading to paralysis and death. In diseases such as ALS, aging is considered a maj...

Deciphering Enhancers of Hearing Loss Genes for Efficient and Targeted Gene Therapy of Hereditary Deafness

Medicine, Neurology, Life Sciences, Bioengineering, Genetics,
hearing loss   gene therapy   hereditary deafness   enhancers   targeted therapy  

Academic Background Hereditary deafness is one of the most common sensory disorders worldwide, affecting over 400 million people, with approximately 60% of congenital deafness linked to genetic factors. Although adeno-associated virus (AAV)-mediated gene therapy shows great potential in treating hereditary deafness, significant concerns remain rega...

High-Efficiency Base Editing in the Retina of Primates and Human Tissues

Medicine, Ophthalmology, Life Sciences, Biochemistry, Bioengineering, Genetics,
base editing   retina   gene therapy   primates   Stargardt disease  

High-precision Base Editing Technology in Primates and Human Retina Research Background Stargardt disease is a currently untreatable, inherited neurodegenerative disease that leads to macular degeneration and blindness due to loss-of-function mutations in the ABCA4 gene. The protein encoded by the ABCA4 gene is a membrane lipid flippase localized i...

In Vivo Expansion of Gene-Targeted Hepatocytes through Transient Inhibition of an Essential Gene

Medicine, Hepatobiliary System, Life Sciences, Bioengineering, Genetics,
gene therapy   hepatocytes   in vivo expansion   essential gene   genome editing  

Breakthrough in Gene Therapy: Repair Drive Technology Enables In Vivo Expansion of Hepatocytes Academic Background Gene therapy has become a hot topic in medical research in recent years, especially for liver diseases. Due to the central role of the liver in metabolism, it has become a critical target for research. Although existing gene-editing te...

Rapid and Scalable Personalized ASO Screening in Patient-Derived Organoids

Medicine, Life Sciences, Cell Biology, Biochemistry, Bioengineering, Genetics,
personalized therapeutics   antisense oligonucleotides   rare diseases   organoid models   gene therapy   Duchenne muscular dystrophy   stem cells  

Establishment of a Personalized Antisense Oligonucleotide (ASO) Screening Platform Based on Patient-Derived Organoids Academic Background In recent years, with the rapid development of genome sequencing technologies, an increasing number of rare genetic diseases have been found to be associated with specific gene mutations. Antisense oligonucleotid...

Gene Therapy: Genetic Engineering of Transfusable Platelets with mRNA-Lipid Nanoparticles is Compatible with Blood Banking Practices

Medicine, Hematology, Life Sciences,
gene therapy   platelets   mRNA-lipid nanoparticles   blood banking   transfusion   cell therapy   clinical compatibility  

Report on the Compatibility of mRNA-Lipid Nanoparticle-Based Gene-Engineered Platelets with Blood Banking Practices Academic Background Platelets play an essential role in hemostasis, inflammation, sepsis, and cancer. However, clinical applications of platelet transfusions are primarily limited to managing thrombocytopenia and bleeding. To broaden ...