Research Trends
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A Study on The Mechanism of Achromatopsia |
Sep 15, 2022 |
Patients with achromatopsia (ACHM) have abnormal color discrimination on the three color vision axes corresponding to the three cone cells. The patient's three cones, which sense red, green and blue, are completely inoperative, so they cannot distinguish between different colors. Read More > |
Newsletter
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Let's Meet at The 3rd Annual Gene Therapy for Ophthalmic Disorders in September! |
Sep 05, 2022 |
The 3rd Annual Gene Therapy for Ophthalmic Disorders meeting is bringing you even more world class content, bringing you two tracks of learning to tackle the pressing challenges faced in the pre-clinical stages and in clinical development. This includes challenges faces with drug targets including wet AMD, dry AMD, DME, IRDs and more. Read More > |
Research Trend
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Mechanisms of Angelman Syndrome and Models for Research |
Sep 01, 2022 |
Angelman syndrome (AS) is a rare brain disorder that affects about 1 in 20,000 newborns. Children with AS don't look much different from normal children on the surface—smiling and gentle. However, behind the angelic smile, there are a number of neurodevelopmental problems. The current treatment strategies for Angelman syndrome mainly focus on gene therapy, and the gene delivered by adeno-associated virus (AAV) vector treatment holds promise as a cure for the disease. Read More > |
Research Trends
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CRISPR-Pro-Mediated Gene Editing System for Effective Treatment of X-linked Retinitis Pigmentosa |
Aug 09, 2022 |
Mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene are associated with X-linked retinitis pigmentosa (XLRP), which accounts for 10% to 20% of all cases of retinitis pigmentosa (RP) and is one of its most severe forms. Herein, we review the research procedure that has culminated in the development of a potential gene therapy for XLRP caused by RPGR mutations. Read More > |
Weekly Gene
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The Culprits of Amyotrophic Lateral Sclerosis (ALS) |
Aug 09, 2022 |
Age and heredity are currently recognized as the most relevant causative factors for amyotrophic lateral sclerosis (ALS), although age is also the biggest risk factor for many other neurodegenerative diseases. While inherited ALS accounts for only 5% of patients, there are more than 20 gene mutations identified as the pathological culprits of ALS. Read More > |
Research Trends
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Why CAR NK Cell Therapy Research Is Quickly Growing |
Aug 05, 2022 |
What is CAR-T? The full name of CAR here is Chimeric Antigen Receptor, its structure includes extracellular domain, transmembrane domain, and an intracellular signal domain. Read More > |
Research Trend
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How to Measure the Viral Titer of Lentivirus Vectors? |
Jun 06, 2022 |
In this article, we will introduce some commonly used lentivirus titer detection methods to assess the quality of the virus and maintain experimental consistency. Read More > |
Research Trend
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Lili Zhou's Team Reveals a New Mechanism of Renal Fibrosis |
May 25, 2022 |
In March 2022, the team of Professor Lili Zhou from Southern Medical University in China published a paper entitled “β-catenin-controlled tubular cell-derived exosomes play a key role in fibroblast activation via the OPN-CD44 axis” in the Journal of Extracellular Vesicles, the top impact factor (IF=25.841) journal in the field of exosome research. Read More > |
Weekly Gene
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Genetic Factors of Eye Disease: Retinitis Pigmentosa (RP) & the RHO Gene |
May 23, 2022 |
Retinitis pigmentosa (RP) is a clinically and genetically heterogeneous blinding retinal disease, which belongs to the large group of inherited retinal degenerations —for which 54 retinal genes have been found to be mutated thus far. Read More > |
Research Trend
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What is Huntington's Disease? |
May 20, 2022 |
Huntington's disease (HD), also known as Huntington's chorea, is a neurodegenerative disease that has a 50% probability of being inherited by children of people living with HD. Read More > |