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Ten Deadly Sins of Rare Diseases
Unraveling Rare Diseases: Researching Hotspot Pathogenic Mutations in Humanized Models
Dec 29, 2023
Welcome to the 'Ten Deadly Sins of Rare Diseases' column, where we decode the pathogenic mechanisms of rare diseases, industry research progress (e.g. gene therapy), and innovative pre-clinical strategies to drive translational research results (such as model construction and drug screening, etc.). Read More >
Ten Deadly Sins of Rare Diseases
Unraveling the Genetic Tapestry of Angelman Syndrome: Decoding Maternal Genetic Abnormalities Behind the 'Happy Puppet' Phenomenon. [Ten Deadly Sins of Rare Diseases Ⅶ]
Nov 24, 2023
Welcome to the 'Ten Deadly Sins of Rare Diseases' Column, where we delve into the intricate world of rare diseases, industry breakthroughs in gene therapy, and innovative pre-clinical research strategies to drive translational successes. In this edition, we shine a spotlight on Angelman Syndrome (AS), a rare disorder linked to the imprinted gene UBE3A. Read More >
Ten Deadly Sins of Rare Diseases
Application of Humanized Mouse Models in DNA Repeat Expansion Diseases [Ten Deadly Sins of Rare Diseases Ⅵ]
Nov 15, 2023
Enter the 'Ten Deadly Sins of Rare Diseases' column to unlock the mechanisms of rare disease occurrence and development, industry research advancements (such as gene therapy), and innovative strategies for advancing clinical outcomes (including model construction and drug screening). Read More >
Ten Deadly Sins of Rare Diseases
Is Smn2 Gene Therapy the Key to Treating Spinal Muscular Atrophy? [Ten Deadly Sins of Rare Diseases Ⅴ]
Oct 20, 2023
In support of August being annually recognized as International SMA Awareness Month, Part V of our "Ten Deadly Sins of Rare Diseases" article series will focus on Spinal Muscular Atrophy (SMA). 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 >
Ten Deadly Sins of Rare Diseases
Exploring RHO-Related Pathogenic Mechanisms and RHO Gene Therapy Research Progress [Ten Deadly Sins of Rare DiseasesⅠ]
Jun 30, 2023
June 6th, 2023 is the 28th National Eye Care Day of China. Do you know the theme of this year's Eye Care Day? Read More >