What a decade of gene therapy research for sickle cell disease taught UCLA scientists
Clinical trial yields insights about the role of chemotherapy, inflammation and trial design that could help shape next-gen curative therapies.
stem cells
Results
FDA approves gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder
All the children treated in a clinical trial are living with restored immune function.
‘Night and day’: Adventuring through childhood with newfound immune systems
After years of hospital stays and fear, families are watching their kids thrive. Today, the gene therapy they received earned accelerated approval from the FDA.
Nanoparticle-based gene editing could expand treatment options for cystic fibrosis
Replacing the entire faulty gene rather than correcting individual mutations may allow the therapy to work across all patients with cystic fibrosis.
UCLA scientists identify optimal design for off-the-shelf immunotherapy targeting solid tumors
The research team engineered human blood stem cells to produce specialized immune cells called NKT cells.
Restoring the healthy form of a protein could revive blood vessel growth in premature infants’ lungs
A UCLA-led research team has discovered a molecular switch that determines whether tiny blood vessels in premature infants’ lungs can regenerate after injury.
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Gene therapy delivers lasting immune protection in children with rare disorder
Long-term follow-up shows 95% success rate, no serious complications in largest and longest ADA-SCID gene therapy study to date.
Headspace invaders: How mosquito-borne viruses breach the brain’s defenses
The findings reveal precise viral-host interactions that could be targeted with new antivirals or vaccines.
UCLA scientists advance gene therapy for deadly blood disorder
In patient cells in culture, the gene therapy successfully restored normal alpha-globin protein production to healthy levels.
UCLA researchers find how epilepsy genes disrupt different brain regions using stem cell models
The new research points to the distinct effects that a single disease-causing gene variation can have across different regions of the brain.
UCLA scientists reprogram stem cells to create renewable cancer-fighting T cells
The clinical trial demonstrates that combining genetically engineered T cells and stem cells can produce cancer-fighting immune cells in humans.
A surgeon’s second calling: Gene therapy for the brain’s energy crisis
How an experimental therapy could transform life for children with creatine transporter deficiency.
Finding confidence through research
Ananya Ravikumar’s passion for finding solutions is leading her to a Ph.D. in gene therapy.
Translational Cell Therapy Lab at UCLA advances cell therapies and trains future scientists
Patients’ own cells could offer interventions for spinal cord injuries, muscular dystrophies and more.
Gene therapy restores immune function and extends lives of children with rare immune disorder
9 children born with severe LAD-l are symptom-free two years after treatment.
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UCLA researchers unite to develop immunotherapy they hope will transform ovarian cancer treatment
A UCLA team is working to create a therapy designed to both eliminate cancer cells and dismantle the tumor’s protective defenses.
UCLA researchers discover a way to make fat cells skinny
Findings could lead to new, more effective therapies for managing diabetes.
Scientists discover ‘entirely unanticipated’ role of protein netrin1 in spinal cord development
Known for its axon guidance properties, new research suggests protein is critical in guiding neural development.
UCLA receives $2 million to develop regenerative medicine therapies for spinal cord injury
The grant from The Marcus Foundation will support research on restoring sensory function and feeling.
Scientists identify ‘missing piece’ required for blood stem cell self-renewal
The study could help make lifesaving stem cell transplants more available.
Sequencing of the developing human brain uncovers hundreds of thousands of new gene transcripts
New study could improve the ability to make genetic diagnoses and treat neurodevelopmental disorders.
Novel technology positions ‘off-the-shelf’ cancer immunotherapy for the clinic
UCLA scientists develop a new method to engineer immune cells that could treat multiple patients.
‘I feel like myself again’: UCLA clinical trial offers hope for lymphoma patients
Hirotaka Matsunaga has been in remission for 18 months after participating in an experimental CAR-T cell therapy led by UCLA professors.