# Event Details

- **Event Name**: How Rare Disease Research is Transforming Medicine Through Gene Therapy
- **Event Start and End Date**: Wed, 19 Aug, 2026 at 07:00 pm – Wed, 19 Aug, 2026 at 08:00 pm
- **Event Description**: How Rare Disease Research is Transforming Medicine Through Gene TherapyAbout this EventResearch on rare genetic disorders has become a powerful driver of innovation in medicine. Dr. Stephanie Cherqui’s research at UC San Diego pioneered a hematopoietic stem cell gene therapy approach for cystinosis, a multisystem lysosomal disorder, establishing a novel therapeutic paradigm in which gene-corrected stem cells can deliver functional organelles to diseased tissues throughout the body. Early clinical studies have shown encouraging safety and efficacy, validating decades of translational research from bench to bedside. Building on these discoveries, her laboratory has expanded this platform to other rare neurodegenerative and metabolic disorders, including Friedreich’s ataxia and Sanfilippo Type C, while uncovering new roles for hematopoietic stem cell–derived immune cells in tissue repair and neuroprotection. These insights are now informing therapeutic strategies for more common conditions, including Alzheimer’s disease, illustrating how research in rare diseases can catalyze transformative advances across medicine.Stephanie Cherqui, UC San DiegoThe National Academies of Sciences, Engineering, and Medicine (NASEM) are committed to the principles of diversity, integrity, civility, and respect in all of our activities. We look to you to be a partner in this commitment by helping us to maintain a professional and cordial environment. All forms of discrimination, harassment, and bullying are prohibited in any NASEM activity. This commitment applies to all participants in all settings and locations in which NASEM work and activities are conducted, including committee meetings, workshops, conferences, and other work and social functions where employees, volunteers, sponsors, vendors, or guests are present.
- **Event URL**: https://allevents.in/irvine/how-rare-disease-research-is-transforming-medicine-through-gene-therapy/100001993552680733
- **Event Categories**: workshops, meetups, science-fair
- **Interested Audience**: 
  - total_interested_count: 0
- **Event Highlights**: 
  - Duration: 1 hour
  - Location: Beckman Center
  - Languages: English

## Ticket Details

- **Ticket Price Range**: min: 0, max: 0, currency: USD

## Event venue details

- **city**: Irvine
- **state**: CA
- **country**: United States
- **location**: Beckman Center
- **lat**: 33.6492861
- **long**: -117.85395010000002
- **full address**: Beckman Center, 100 Academy Way, Irvine, United States

## Event gallery

- **Alt text**: How Rare Disease Research is Transforming Medicine Through Gene Therapy
  - **Image URL**: https://cdn-az.allevents.in/events1/banners/df212ab3a3e3641eaa694413a2d2b88cce74a091eb5aa25cfaea8d6394a5f3b3-rimg-w451-h112-dc040403-gmir.jpg?v=1783665970

## FAQs

- **Q**: When and where is How Rare Disease Research is Transforming Medicine Through Gene Therapy being held?
  - **A:** How Rare Disease Research is Transforming Medicine Through Gene Therapy takes place on Wed, 19 Aug, 2026 at 07:00 pm to Wed, 19 Aug, 2026 at 08:00 pm at Beckman Center, 100 Academy Way, Irvine, United States.
- **Q**: Who is organizing How Rare Disease Research is Transforming Medicine Through Gene Therapy?
  - **A:** How Rare Disease Research is Transforming Medicine Through Gene Therapy is organized by Distinctive Voices.
- **Q**: Who is this event for? Is it right for me?
  - **A:** How Rare Disease Research is Transforming Medicine Through Gene Therapy is ideal for curious learners, students, and skill-builders looking to gain hands-on knowledge and practical expertise in a focused, interactive setting. Whether you're a first-time attendee or a longtime enthusiast in Irvine, this event is thoughtfully curated to deliver a standout experience worth every moment. If How Rare Disease Research is Transforming Medicine Through Gene Therapy sounds like your kind of event, don't wait - spots fill up fast.

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