Cell and gene therapy promise to repair Parkinson's brain

After decades of pre-clinical development, the cell and gene therapy for Parkinson's disease (PD) are currently being actively explored

"The long-term mission of cell and gene therapy for brain diseases, from the first speculative ideas and the first exploratory experiments in the 1980s and 1990s, was the main setback in the field of cell and gene therapy at the turn of the century, followed closely.

"The renaissance in the field of cell and gene therapy is driven by significant technological developments that have allowed us to obtain more and more powerful molecular and genetic tools and technologies

Pioneering trials in this field face regulatory and ethical challenges that are very different from traditional drug trials

This article reviews the use of advanced therapies (ATMPs), including cell and gene therapy, from the perspective of researchers and patients

For the patient, a reasonable definition of an ideal advanced treatment is an address of one or more patients with unmet needs that are not affected by the existing therapies, treatment and novel mechanisms of action, and most importantly, this intervention is possible Become a disease modification, according to two patient advocates/scientists, Dr.

Past invasive procedures have proved that participating in clinical trials and clinical use of advanced therapies has its own risks higher than standard therapies

"The ethical and regulatory issues associated with these more advanced treatment modalities are completely different from those involving standard drug trials because they must allow for the gradual optimization and refinement of products, dosages, and administration," Anders Björklund, PhD, Wallenberg Neuroscience Center, Lund University, Sweden

The article in the second part of this supplement emphasizes research that promotes the further development of cell and gene therapy

Dr.

Many potential pathogenic and risk-related genes of PD are related to lysosomal function or lysosomal transport, indicating that lysosomal dysfunction is a common feature of PD disease pathology

"The next breakthrough in the treatment of Parkinson's disease will be based on interventions that prevent disease progression and restore function