Genome Editing VEGFA Prevents Corneal Neovascularization In Vivo

Corneal neovascularization (CNV) is a common clinical finding seen in a range of eye diseases. Current therapeutic approaches to treat corneal angiogenesis, in which vascular endothelial growth factor (VEGF) A plays a central role, can cause a variety of adverse side effects. The technology of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 can edit VEGFA gene to suppress its expression. CRISPR offers a novel opportunity to treat CNV.

A model of human neural networks reveals NPTX2 pathology in ALS and FTLD

Human cellular models of neurodegeneration require reproducibility and longevity, which is

PCDHA9 as a candidate gene for amyotrophic lateral sclerosis

There has been new progress in the research of Amyotrophic Lateral Sclerosis (ALS), with the discovery of a novel pathogenic gene—PCDHA9. Mouse animal models have confirmed that mutations at this pathogenic site can lead to a typical ALS phenotype, and an in-depth analysis of the pathogenic mechanism has been conducted.

Lancet | Fudan University Team: The World's First Clinical Trial with Dual AAV Vectors

Today (January 25th), the Eye, Ear, Nose, and Throat Hospital affiliated with Fudan University took

Research | Academician Xianqun Fan's Team at Shanghai Jiao Tong University: Progress and Prospects of AAV in Gene Therapy Applications for Eye Diseases

Recently, the team led by Academician Fan Xianqun from Shanghai Jiao Tong University published a r

Nature Biotechnology: Another masterpiece from a Nobel laureate! Doudna's team develops a new method to deliver genome editing tools to specific cells.

ViruseOn January 14, 2024, the team led by Jennifer A. Doudna at the University of California, Berkeley, published a research paper titled "In vivo human T cell engineering with enveloped delivery vehicles" in Nature Biotechnology online.

Co-transplantation of autologous Treg cells in a cell therapy for Parkinson's disease

Together, these data emphasize the importance of the initial inflammatory response to surgical injury in the differential survival of cellular components of the graft, and suggest that co-transplanting autologous Treg cells effectively reduces the needle-trauma-induced death of mDANs, providing a potential strategy to achieve better clinical outcomes for cell therapy in Parkinson's disease.

CRISPR-assisted transcription activation by phase separation proteins

These findings demonstrate the feasibility of using phase-separation proteins to assist in the regulation of gene expression and support the broad appeal of the dCas9-VPRF system in basic and clinical applications.

Targeting L-selectin Lymphocytes to Deliver Immunosuppressive Drug in Lymph Nodes for Durable Multiple Sclerosis Treatment

Abstract     Inflammation induced by autoreactive CD4+ T lymphocytes is a major factor in the path

Advances in gene therapy hold promise for treating hereditary hearing loss

Herein, we address three main gene therapy strategies (gene replacement, gene suppression, and gene editing), summarizing the strategy that is most appropriate for particular monogenic diseases based on different pathogenic mechanisms, and then focusing on their successful applications for HHL in preclinical trials. Finally, we elaborate on the challenges and outlooks of gene therapy for HHL

CRISPR-assisted transcription activation by phase-separation proteins

These findings demonstrate the feasibility of using phase-separation proteins to assist in the regulation of gene expression and support the broad appeal of the dCas9-VPRF system in basic and clinical applications.

The landscape of tolerated genetic variation in humans and primates

We show that these variants can be inferred to have nondeleterious effects in humans based on their presence at high allele frequencies in other primate populations. We use this resource to classify 6% of all possible human protein-altering variants as likely benign and impute the pathogenicity of the remaining 94% of variants with deep learning, achieving state-of-the-art accuracy for diagnosing pathogenic variants in patients with genetic diseases.

Nature: AI constructs gene interaction networks to accelerate discovery of disease treatment targets

This study generated a gene expression dataset - Genecorpus-30M, which includes about 30 million single cell transcriptomic data from various human tissues. The research team pre-trained an AI model based on transfer learning - Geneformer, using this dataset to enable prediction of gene network dynamics, mapping of gene networks, and accelerated discovery of disease treatment candidate targets in limited data.