Breakthrough In Vivo Gene Therapy Offers Hope for Parkinson's Disease Sufferers

The video discusses the first in vivo gene therapy for Parkinson's disease, led by Dr. Michael Kaplit. The procedure involves infusing viral particles with a human gene to produce GABA, aiming to relieve symptoms. Nathan Klein, the first patient, shared his positive experience post-procedure. The trial is in phase one, focusing on safety. Despite FDA approval, some experts criticize the lack of prior evidence in monkeys. Klein was awake during the surgery, which involved drilling into his skull. Current medical therapies often become ineffective or cause side effects over time.

What is the primary goal of the viral particles infused during the gene therapy procedure?

• To destroy nerve cells causing Parkinson's

• To produce a protein that helps alleviate symptoms

• To stimulate the immune system

• To replace damaged brain tissue

What is the main objective of the phase one clinical trial for this gene therapy?

• To determine the efficacy of the treatment

• To assess the safety of the procedure

• To compare it with other treatments

• To test it on a large population

How many patients were approved by the FDA for this gene therapy trial?

• 12

• 10

• 15

• 20

What was the most uncomfortable part of the procedure for Nathan Klein?

• The recovery period

• The press conference

• The drilling of a small hole in his skull

• The infusion of viral particles

What concern did experts raise about the gene therapy procedure?

• It was too expensive

• It lacked evidence from animal studies

• It was not approved by the FDA

• It had severe side effects