Beijing Ruicy Gene Therapy Institute for Rare Diseases, commonly referred to as RDGT Institute, was formally established in September 2014 as a non-profit scientific research organization registered in Beijing under the Bureau of Civil Affairs. The Institute operates as a private non-enterprise entity dedicated to the public good in both science and industry, focusing primarily on advancing the development of gene therapies for rare diseases and harnessing scientific advancements to serve the rare disease community. As the pioneering non-profit research entity in China to concentrate exclusively on gene therapy for rare ailments, the RDGT Institute is committed to conducting basic research in gene therapy for rare diseases, developing related pharmaceuticals, and facilitating the progression of these treatments into clinical trials.

100 Clinical Results associated with Beijing Ruicy Gene Therapy Institute for Rare Diseases

0 Patents (Medical) associated with Beijing Ruicy Gene Therapy Institute for Rare Diseases

Literatures (Medical) associated with Beijing Ruicy Gene Therapy Institute for Rare Diseases

01 Aug 2022·Current Gene TherapyQ4 · MEDICINE

AAV9-coGLB1 Improves Lysosomal Storage and Rescues Central Nervous System Inflammation in a Mutant Mouse Model of GM1 Gangliosidosis

Q4 · MEDICINE

Article

Author: Tang, Fang ; Feng, Yuyu ; Ma, Wenhao ; Liu, Sichi ; Jia, Xuefang ; Wu, Xiaobing ; Tang, Chengfang ; Zhang, Yan ; Huang, Yonglan

Background:GM1 gangliosidosis (GM1) is an autosomal recessive disorder characterized by the deficiency of beta-galactosidase (β-gal), a ubiquitous lysosomal enzyme that catalyzes the hydrolysis of GM1 ganglioside.Objective:The study aims to explore the application of the AAV9-coGLB1 for effective treatment in a GM1 gangliosidosis mutant mouse model.Methods:We designed a novel adeno-associated virus 9 (AAV9) vector expressing β-gal (AAV9- coGLB1) to treat GM1 gangliosidosis. The vector, injected via the caudal vein at 4 weeks of age, drove the widespread and sustained expression of β-gal for up to 32 weeks in the Glb1G455R/G455R mutant mice (GM1 mice).Results:The increased levels of β-gal reduced the pathological damage occurring in GM1 mice. Histological analyses showed that myelin deficits and neuron-specific pathology were reduced in the cerebral cortex region of AAV9-coGLB1-treated mice. Immunohistochemical staining showed that the accumulation of GM1 ganglioside was also reduced after gene therapy. The reduction of the storage in these regions was accompanied by a decrease in activated microglia. In addition, AAV9 treatment reversed the blockade of autophagic flux in GM1 mice.Conclusion:These results show that AAV9-coGLB1 reduces the pathological signs of GM1 gangliosidosis in a mouse model.

Scientific ReportsQ3 · CROSS-FIELD

Deletion of the B-B’ and C-C’ regions of inverted terminal repeats reduces rAAV productivity but increases transgene expression

Q3 · CROSS-FIELD

ArticleOA

Author: Dong, Xiaoyan ; Wu, Xiaobing ; Tian, Wenhong ; Zhou, Qingzhang ; Liu, Chunguo ; Lian, Zhonghui

AbstractInverted terminal repeats (ITRs) of the adeno-associated virus (AAV) are essential for rescue, replication, packaging, and integration of the viral genome. While ITR mutations have been identified in previous reports, we designed a new truncated ITR lacking the B-B’ and C-C’ regions named as ITRΔBC and investigated its effects on viral genome replication, packaging, and expression of recombinant AAV (rAAV). The packaging ability was compared between ITRΔBC rAAV and wild-type (wt) ITR rAAV. Our results showed the productivity of ITRΔBC rAAV was reduced 4-fold, which is consistent with the 8-fold decrease in the replication of viral genomic DNA of ITRΔBC rAAV compared with wt ITR rAAV. Surprisingly, transgene expression was significantly higher for ITRΔBC rAAV. A preliminary exploration of the underlying mechanisms was carried out by inhibiting and degrading the ataxia telangiectasia mutated (ATM) protein and the Mre11 complex (MRN), respectively, since the rAAV expression was inhibited by the ATM and/or MRN through cis interaction or binding with wt ITRs. We demonstrated that the inhibitory effects were weakened on ITRΔBC rAAV expression. This study suggests deletion in ITR can affect the transgene expression of AAV, which provides a new way to improve the AAV expression through ITRs modification.

100 Deals associated with Beijing Ruicy Gene Therapy Institute for Rare Diseases

100 Translational Medicine associated with Beijing Ruicy Gene Therapy Institute for Rare Diseases

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Beijing Ruicy Gene Therapy Institute for Rare Diseases

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