>. Insufficient β-globin production affects erythropoiesis, and the destruction of red blood cells leads to chronic hemolytic anemia and compensated extramedullary hematopoiesis
Express FDA release, Pfizer DMD gene therapy phase 3 clinical trial is about to restart
Time of Update: 2022-05-30
S. Food and Drug Administration (FDA) has notified the company to allow its Phase 3 clinical trial of fordadistrogene movaparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), to continue .
Gene therapy to reverse autism-related gene mutations in brain organoids and their effects on neurodevelopment
Time of Update: 2022-05-27
To study the impact of TCF4 gene mutations on brain development, the research team focused on Pitt-Hopkins syndrome, a form of autism caused by mutations in the TCF4 gene, in children with severe cognitive and motor impairments .
full sequence analysis of De Novo and rare gene variants in patients with cerebral arteriovenous malformations
Time of Update: 2022-05-26
Other interesting genetic studies with smaller sample sizes have also identified several candidate genes for bAVM through triplet/family analysis or sequencing of individual cases .
Liu Lijun Ren Ruizheng Head of Gene Biology Department
Time of Update: 2022-05-26
Lijun Liu will be responsible for the analysis, testing and evaluation of the company's gene editing products in the preclinical and clinical stages, and participate in the project establishment of the products .
Takeda Announces Collaboration to Develop Reproducible, Non-Viral Gene Therapies
Time of Update: 2022-05-23
drug delivery platform to design and develop treatments for rare diseases Indications for gene therapy . According to the press release, the potential total transaction value of the partnership
Cell and gene therapy represented by CAR-T therapy has become a prairie prairie
Time of Update: 2022-05-23
? Standardized development stage (2016-present) In 2017, the State Food and Drug Administration promulgated the "Technical Guidelines for Research and Evaluation of Cell Therapy Products (Trial)", which proposed the safety, Valid, quality-controllable general technical requirements .
Code Announces R&D Agreement with Takeda to Develop Non-Viral Vector Gene Therapies
Time of Update: 2022-05-23
References:[1] Code Biotherapeutics Announces Collaboration with Takeda to Use Proprietary 3DNA Genetic Medicine Delivery Platform to Design and Develop Gene Therapies for Rare Diseases.
Voyager and Novartis reach over $1.7 billion partnership to develop next-generation AAV gene therapy
Time of Update: 2022-05-23
References:[1] Voyager Therapeutics Announces License Option Agreement with Novartis for Target-Specific Access to Next-Generation TRACER™ AAV Capsids for Gene Therapy Programs.
Innovative CRISPR technology discovers genes that alter the tumor microenvironment and is expected to provide new anti-cancer targets
Time of Update: 2022-05-22
Combining in situ CRISPR screening with spatial transcriptomics, researchers at the Icahn School of Medicine at Mount Sinai in New York have identified genes that control lung cancer growth, respond to immunotherapy, and already regulate the tumor microenvironment.
Astellas' gene therapy takes another hit
Time of Update: 2022-05-22
AT702 is an AAV antisense gene therapy designed for DMD patients with exon 2 skipping .
Study reveals molecular mechanism underlying altered gene expression in the brains of Alzheimer's patients
Time of Update: 2022-05-22
Now, in a study titled "Multi-transcriptomic analysis points to early organelle dysfunction in human astrocytes in Alzheimer's disease" published in the international journal Neurobiology of Disease, scientists from Spain analyzed data from nearly 800 deceased individuals.
Xu Shuhua's team released the ArchaicSeeker2.0 method to reconstruct the analysis process of the ancient human gene communication model
Time of Update: 2022-05-20
)" and other funds . >Paper link: https://star-protocols. cell. com/protocols/1572> > >
Link between Parkinson's genes and voice?
Time of Update: 2022-05-20
A unique and ideal model for studying human languageTo study the relationship between vocal changes and a Parkinson's disease-related gene called alpha synuclein, the researchers turned to the zebra finch, a native Australian songbird .
Green Bamboo Bio uses Lonza GS Xceed(R) gene expression system to develop recombinant shingles vaccine and bispecific antibody
Time of Update: 2022-05-20
transcriptional activity . >https://pharma. lonza. com/offerings/specialized-capabilities/expressions-technologies/GS-piggyBac >
Deletion of a single allele of the gene encoding Zbtb38 causes early embryonic death
Time of Update: 2022-05-19
"Our results show that germline deletion of Zbtb38 monoallelic reduces ectodermal cell proliferation shortly after embryo implantation and increases apoptosis, leading to early embryonic death .
relatives" based on some genes and phenotypes is not reliable
Time of Update: 2022-05-18
Building species relationship trees based on partial genes and phenotypes is unreliableTo understand the impact of incomplete lineage splitting on the evolution of species morphology and traits, the researchers in the paper used marsupials .
AACR Blockbuster: Combination of high gene amplification and tumor mutational burden as a pan-cancer biomarker in patients receiving tislelizumab
Time of Update: 2022-05-18
A high tumor mutational burden (TMB-H) was associated with elevated neoantigen expression in tumors and increased response to PD-(L)1 inhibitors .
