NeurologyLive Brain Games: A weekly neurology trivia spotlight
NeurologyLive® Brain Games is a weekly quiz series designed for clinicians, researchers, and neurology enthusiasts. Fresh questions go live every Sunday morning and challenge you across a spectrum of topics—from dementia and epilepsy to stroke, sleep disorders, and the cutting edge of gene therapy. This week’s theme centers on Duchenne muscular dystrophy (DMD) and the gene therapy landscape, offering a concise, clinically relevant way to stay sharp while exploring new therapeutic strategies.
Theme preview: Duchenne muscular dystrophy and gene therapy
For the October 12, 2025 edition, the questions focus on the therapeutic goals and delivery systems behind modern DMD gene therapies, with emphasis on micro-dystrophin constructs and their clinical implications. These topics reflect real-world progress in neuromuscular medicine and the broader intersections with neurology, rehabilitation, and patient advocacy.
Question 1: Therapeutic goal of micro-dystrophin gene therapy in DMD
Question: Which of the following best describes the therapeutic goal of micro-dystrophin gene therapy in Duchenne muscular dystrophy?
- Replacement of the full-length dystrophin gene to restore complete muscle function
- Delivery of a truncated dystrophin gene capable of producing a functional dystrophin-like protein
- Stimulation of satellite cell proliferation through AAV-mediated signaling
- Reduction of inflammation through inhibition of NF-κB signaling
Answer: Delivery of a truncated dystrophin gene capable of producing a functional dystrophin-like protein. This approach uses truncated, or micro, dystrophin to fit into delivery vectors while aiming to restore key dystrophin functions in muscle tissue. It’s a strategic balance between gene size, vector capacity, and therapeutic efficacy, with ongoing research to optimize expression and clinical outcomes.
Question 2: Vector system for DMD gene therapies
Question: Which vector system is most commonly used for gene delivery in approved or investigational DMD gene therapies?
- Lentivirus
- Adenovirus
- Adeno-associated virus
- Retrovirus
Answer: Adeno-associated virus (AAV). AAV vectors are favored in many DMD gene therapy programs due to their relatively favorable safety profile, ability to transduce muscle tissue, and non-integrating nature. Researchers continue to refine serotypes and dosing strategies to maximize muscle delivery while minimizing immune responses.
Question 3: Immune-related hepatotoxicity monitoring after gene therapy
Question: Which laboratory finding is most closely monitored post-gene therapy administration to assess for potential immune-related hepatotoxicity?
- Creatine kinase (CK)
- Gamma-glutamyl transferase (GGT)
- Alanine aminotransferase (ALT)
- Troponin I
Answer: Alanine aminotransferase (ALT). ALT elevation can indicate hepatotoxicity and immune-mediated liver injury, which necessitates careful monitoring after gene delivery. Clinicians often pair ALT tracking with clinical assessments and, when indicated, other hepatic panels to ensure patient safety during the therapeutic window.
How did you do? Join the conversation
We’d love to hear how you scored on this week’s Brain Games quiz. Share your results and compare with peers in your network by replying to the poll or discussion thread where this quiz is posted. These weekly brain teasers are meant to be educational, engaging, and a community-building exercise for clinicians and patients alike.
Why Brain Games matter for clinicians
Beyond testing knowledge, Brain Games encourages clinicians to stay current on evolving treatment modalities, such as Duchenne gene therapy, and to consider their practical implications in patient care—ranging from eligibility and monitoring to long-term outcomes and advocacy. The questions invite you to think critically about vector choices, therapeutic endpoints, and safety monitoring in real-world settings.
Submit your questions and stay in the loop
Interested in contributing questions? Reach out to the Brain Games editor, Marco Meglio, to help expand the weekly quiz library and keep the content fresh and clinically relevant. Submissions help keep the series aligned with the latest advances in neurology and neuromuscular medicine.