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University of California, Los Angeles

11000 Kinross Avenue
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Los Angeles, CA 90095-1406
United States

Optimizing Novel Strategies for Lipid Nanoparticle Mediated CRISPR Gene Editing Towards CAR T Cell Therapies

Acute Lymphoblastic Leukemia (ALL)

Mentor Name: Ruby Sims

Arjun Gupta

University of California, Los Angeles

Enabling lipid nanoparticle gene therapy treatments for fusion positive alveolar rhabdomyosarcoma through enhanced stealth and tumor targeting modifications

Rhabdomyosarcoma

Mentor Name: Steven Jonas

Kody Seow

University of California, Los Angeles

In vivo transdermal generation of CD33 CAR T-cells towards acute myeloid leukemia

Leukemia, Acute Myeloid Leukemia (AML)

Pediatric acute myeloid leukemia (AML) is the second most common pediatric leukemia, with treatments still based on intense, conventional chemotherapy and overall survival remaining below 70%. Recently, therapeutic research has explored the engineering of a patient’s own immune cells to target unique AML cell surface markers. However to manufacture these therapies immune cells must be collected from patients whose immune system has been depleted by multiple rounds of chemotherapy.

Amadu Tadesse

University of California, Los Angeles

Itzel Huerta

University of California, Los Angeles

Steven Jonas, MD/PhD

University of California, Los Angeles

Dissecting the disease evolution of myeloid leukemia of Down Syndrome using iPSC models

Leukemia

We aim to createa disease-in-a-dish model to understand myeloid leukemia in children with Down syndrome (ML-DS). Trisomy 21 (T21), or Down syndrome (DS), is a genetic condition caused by an extra copy of chromosome 21. This leads to developmental differences, including distinct blood cell abnormalities. Young children with DS have a higher risk of developing blood cancers, which begins with a pre-leukemic condition, transient abnormal myelopoiesis (TAM), before birth, after acquiring a genetic change in the GATA1 gene. Later it can develop into leukemia if more mutations arise.

Designing targeted lipid nanoparticles for CAR T cell generation

Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL)

Mentor Name: Ruby Sims

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Featured Hero

Sawyer Knoblock

Sawyer is a young man with a big personality who always shows so much love for others. When he was diagnosed with germinoma, treatment had to be tailored specifically to him since he also has Down syndrome. Luckily, it worked and today Sawyer is NED!
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