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Research Trends
Rare Diseases Are Not Rare: The Mechanistic Research on Kennedy Disease (KD)
Sep 22, 2023
Kennedy disease (KD), also known as Spinal and Bulbar Muscular Atrophy (SBMA), is a recessive, X-linked, adult-onset motor neuron disease characterized by the slow, progressive weakness of the bulbar and limb muscles. Kennedy disease predominantly affects adult males, with an estimated global incidence of 1-2/100,000. Read More >
Research Trends
Do not give up chasing the light—Gene therapy aids the blind in regaining their sight
Sep 19, 2023
It is said that the eyes are the windows to the soul, but there are many people with congenital blindness who cannot "see" the light. According to publicly available data from the World Health Organization, there are at least 2.2 billion people globally with impaired vision or blindness. Read More >
Research Trends
Exploring the Power of the Cre-Lox System: Conditional Gene Targeting in Mice for In Vivo Applications
Sep 08, 2023
Conditional gene targeting in mice has revolutionized the field of molecular biology and genetic engineering. In vivo applications of the Cre-lox system are highly efficient, specific, and versatile. The Cre-lox system is a powerful tool to manipulate and study gene function in vivo, and it relies on the Cre recombinase enzyme to induce site-specific recombination between two LoxP sites. Read More >
Research Trends
What is Familial Exudative Vitreoretinopathy (FEVR)? What are the mouse models for it?
Aug 31, 2023
Familial Exudative Vitreoretinopathy (FEVR) is a group of rare hereditary retinal disorders characterized by abnormal retinal vascular development, leading to incomplete peripheral retinal vascularization and subsequent retinal ischemia. Read More >
Research Trends
Can Childhood Metabolic Disorders Actually Lead to Heart Failure? Gene Therapy Research Provides Hope
Aug 18, 2023
Have you ever encountered patients who, despite growing normally during early childhood, gradually begin to exhibit symptoms such as facial abnormalities, growth retardation, delayed intellectual development, skeletal deformities, neurological abnormalities, and cardiac involvement? Read More >
Research Trends
Recommended Classic Mouse Models for Metabolic and Cardiovascular Diseases! Double Assurance with Validation Data and Efficacy Evaluation System
Aug 11, 2023
Cardiovascular diseases are now the leading cause of death worldwide, posing a significant threat to human health. In China alone, there are approximately 330 million people with cardiovascular disease and 450 million with metabolic disease. Read More >
Latest Discovery
Igniting Momentum: GCT Industry Rekindles in 2023, Poised for Success
Jul 28, 2023
According to the latest quarterly report from ASGCT (American Society of Gene & Cell Therapy) , Q2 2023 proved to be a notable quarter in the field of therapy approvals, with the introduction of six new therapies that garnered regulatory clearance. Read More >
Ten Deadly Sins of Rare Diseases
Reviewing Muscular Dystrophy: Types, Pathology, and Gene Therapy Research [Ten Deadly Sins of Rare Diseases Ⅳ]
Jul 24, 2023
Step into the 'Ten Deadly Sins of Rare Diseases' series, where we unlock the mechanisms of occurrence and development of rare diseases, research advancements in the field of gene therapy, and innovative preclinical strategies related to model development and drug screening that drive translation of outcomes. Read More >
Ten Deadly Sins of Rare Diseases
The TARDBP gene is closely associated with pathogenesis of amyotrophic lateral sclerosis (ALS), but is it an angel or a demon? [Ten Deadly Sins of Rare Diseases Ⅲ]
Jul 14, 2023
Step into the 'Ten Deadly Sins of Rare Diseases' column, where we unravel the mechanisms underlying the occurrence and development of rare diseases, delve into gene therapy advancements, and explore innovative preclinical model development and drug screening strategies to drive effective translational research outcomes. Read More >
Ten Deadly Sins of Rare Diseases
Why are humanized mice more suitable for hemophilia research, which has a higher prevalence in men? [Ten Deadly Sins of Rare Diseases Ⅱ]
Jul 07, 2023
Rare diseases have always been a big challenge in the field of medical science. Noy only do these diseases have complex mechanisms of occurrence and different types, but importantly, there are no suitable animal models for studying the underlying pathogenesis and evaluating efficacy of drugs. Read More >