MVX 220: A Gene Replacement Therapy for Angelman Syndrome

by James M Wilson, MD, PhD, GEMMABio and Elizabeth Berry-Kravis, MD, PhD, Rush Medical Center

What Does “In Vitro” Mean?
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What Does “In Vitro” Mean?

Frontotemporal Dementia - Diagnostic Overview (H. Branch Coslett)
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Frontotemporal Dementia - Diagnostic Overview (H. Branch Coslett)

AERD: Aspirin Challenge, Desensitization Protocols, and Outcomes - Dr. John Bosso
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AERD: Aspirin Challenge, Desensitization Protocols, and Outcomes - Dr. John Bosso

Will Australia's social media ban for under-16s work? - The Global Story podcast, BBC World Service
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Will Australia's social media ban for under-16s work? - The Global Story podcast, BBC World Service

Quantum Consciousness and the Origin of Life
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Quantum Consciousness and the Origin of Life

A Novel Path to the Brain: Crossing the Blood Brain Barrier for Gene Therapies
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A Novel Path to the Brain: Crossing the Blood Brain Barrier for Gene Therapies

'This Isn't Normal, And It Isn't Pain-Free': Luke Rosen's Parenting Journey
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'This Isn't Normal, And It Isn't Pain-Free': Luke Rosen's Parenting Journey

Hop Topics in Heat & Health: Heat and Children's Health
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Hop Topics in Heat & Health: Heat and Children's Health

Od Wielkiego Wybuchu, przez Dinozaury, po Człowieka - Historia Życia na Ziemi! [Podcast Historyczny]
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Od Wielkiego Wybuchu, przez Dinozaury, po Człowieka - Historia Życia na Ziemi! [Podcast Historyczny]

What do tech pioneers think about the AI revolution? - The Engineers, BBC World Service
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What do tech pioneers think about the AI revolution? - The Engineers, BBC World Service

Telephone physical activity coaching in populations with walking limitations - Dr Louise Pearce
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Telephone physical activity coaching in populations with walking limitations - Dr Louise Pearce

Trump Preps for 80th Birthday, Threatens to Hit Iran, Knicks Historic Win & Elon Musk Trillionaire!?
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Trump Preps for 80th Birthday, Threatens to Hit Iran, Knicks Historic Win & Elon Musk Trillionaire!?

How ASML Makes Chips Faster With Its New $400 Million High NA Machine
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How ASML Makes Chips Faster With Its New $400 Million High NA Machine

Peripheral and Autonomic Neuropathy in ATTR Amyloidosis
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Peripheral and Autonomic Neuropathy in ATTR Amyloidosis

Diabetes Management in 2026
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Diabetes Management in 2026

Angelman Syndrome - Gene therapy
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Angelman Syndrome - Gene therapy

CRG-AS-001: A Non-Viral Delivered CRISPR Gene Editing Approach for AS: From Bench to Bedside
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CRG-AS-001: A Non-Viral Delivered CRISPR Gene Editing Approach for AS: From Bench to Bedside

Overview of Frontotemporal Lobar Degeneration
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Overview of Frontotemporal Lobar Degeneration

An Update on Anti-Amyloid Treatments for Alzheimer's Disease
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An Update on Anti-Amyloid Treatments for Alzheimer's Disease

Something is jamming GPS over Europe. Here's what we found
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Something is jamming GPS over Europe. Here's what we found