Understanding ALS: The Role of Gene Therapy in Treating Amyotrophic Lateral Sclerosis
"Amyotrophic lateral sclerosis (ALS)" is commonly known as "Lou Gehrig's disease." It is a rare neurodegenerative disease and is considered one of the top five incurable diseases in the world. Due to the gradual loss of neurons in the brain, brainstem, and spinal cord...
How to Optimize the Design of AAV and Lentivirus Transfer Plasmids
Designing, constructing, and managing viral vector plasmids can be challenging and time-consuming. Researchers often encounter several hurdles, but with the right support, these challenges can be overcome. Here, we explore common issues and how PackGene's vector...
Digest: Gene Editing Therapeutics Based on mRNA Delivery
Overview This review article discusses the therapeutic potential of gene editing, focusing on mRNA-based delivery systems. It highlights the advancements in gene editing technologies like CRISPR-Cas systems and base editors, and examines their applications in treating...
Lou Gehrig’s Disease: Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis (ALS), commonly known as "Lou Gehrig's disease," is a rare neurodegenerative condition and one of the world's five major incurable diseases. It is characterized by the gradual loss of neurons in the brain, brainstem, and spinal cord that...
Innovative Glaucoma Treatment Strategy Developed by Sun Yat-sen University and University of Cambridge Team
Glaucoma is a multifactorial disease characterized by the gradual apoptosis and degeneration of retinal ganglion cells (RGCs), leading to corresponding vision loss and being one of the leading causes of irreversible blindness worldwide. Elevated intraocular pressure...
A Comprehensive Guide to AAV-Based Gene Therapy in Neurological Disorders: A Nature Review Digest
We recently came across a comprehensive review in Nature Review Drug Discovery by Ling, Q. et al., which provides in-depth insights into AAV-based gene therapy, particularly for neurological disorders. In this article, we'd like to share a concise summary of the key...
Differences in AAV Administration Routes in Clinical Applications for Neurological Diseases
Gene therapy has been a focal point in the biopharmaceutical industry over the past five years. Initially considered for the treatment of rare genetic diseases, gene therapy has gradually been applied to neurological diseases such as Alzheimer's and Parkinson's due to...
Strategies for AAV Applications in the Nervous System
Adeno-associated virus (AAV) has many advantages such as small size, high safety, and low immunogenicity, making it widely used in the study of nervous system diseases. What key points should be considered for AAV applications in neural tissues? Selection of...
Innovations in AAV Production: Leveraging High-Yield Cell Lines, novel RC plasmid, and Dual-Plasmid Systems
Abstract PackGene has pioneered innovative approaches to meet the escalating demand for high-quality adeno-associated virus (AAV) vectors across the dynamic landscape of gene therapy. For example, PackGene achieves remarkable enhancements in AAV packaging efficiency...
The principles and advantages of constructing self-complementary double-stranded AAV (scAAV)
Recombinant adeno-associated virus (rAAV) serves as a gene delivery vector in vivo, where the necessary condition for transcription to proceed is the conversion of the genome sequence from single-stranded AAV (ssAAV) to double-stranded AAV. When it comes to the...
AAV Serotype Screening System
Recombinant adeno-associated viruses (rAAV) are the most commonly used delivery vectors in gene therapy. However, naturally occurring AAVs with organ-targeting properties are very limited. In order to achieve more precise treatment goals, scientists have continuously...
Adeno-associated virus (AAV) Packaging: A Comprehensive Exploration of its Packaging, Mechanisms, and Diverse Applications
Adeno-associated virus (AAV) is a member of the Parvoviridae family, a type of non-enveloped icosahedral virus with a diameter of approximately 20-26 nm and containing around 4.7 kb of linear single-stranded DNA as its genome. Recombinant adeno-associated virus (rAAV)...
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