A Global Alliance to Save Lives: How Two Parents and PackGene Are Redefining What’s Possible in Gene Therapy with Help from Weill Cornell Medicine
HOUSTON, TX – Two Australian parents, faced with the unimaginable prospect of losing their 14-month-old daughter to the ultra-rare neurological disease Hereditary Spastic Paraplegia Type 56 (SPG56), sold their home and everything they owned to save their daughter....
NeuExcell’s NXL-004 Secures FDA Orphan Drug Designation for Glioma Treatment
On December 7, 2023, Beijing time, NeuExcell Therapeutics announced that its proprietary AAV gene therapy product, NXL-004, designed for treating malignant glioma, has been awarded Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). This...
Tauopathy’s Role in Aβ Oligomer Formation: New Insights from Transgenic Primate Models on Alzheimer’s Pathogenesis
Tauopathy, characterized by protein tau and Aβ oligomer accumulation, is a significant aspect of Alzheimer's disease. The causal relationship between these accumulations and neurodegeneration remains unclear despite their links to various cognitive and movement...
Targeted Lysosomal Degradation of Mutant Huntingtin by Engineered Intrabody Peptide SM3
Huntington's disease (HD) is a devastating neurodegenerative condition caused by a mutation in the Huntingtin gene (HTT). This leads to the production of a mutant form of the huntingtin protein (mHTT) with an extended polyglutamine (polyQ) repeat, which has a...
Researchers Unveil a Novel Strategy for Protein Miniaturization in the CRISPR-Cas System
In a groundbreaking study published in Nature Communications, a team of researchers from China have introduced an innovative approach to protein miniaturization within the gene-editing realm of the CRISPR-Cas system. The CRISPR-Cas system is celebrated for its...