Cell & Gene Therapy
Researchers are developing cell and gene therapies to heal or replace damaged tissue and organs and address the root causes of a range of devastating conditions.
Overview
Cell and gene therapies are transforming how we treat a host of diseases that were once thought to be incurable. Our scientists are committed to unlocking the enormous potential of this new class of “living drugs” by translating their foundational knowledge about stem cellsCells that have the ability to differentiate into multiple types of cells and make an unlimited number of copies of themselves.stem cellsCells that have the ability to differentiate into multiple types of cells and make an unlimited number of copies of themselves. and human development into life-saving therapies. Their work is already resulting in successful treatments in clinical trials and more therapies in the pipeline for conditions ranging from sickle cell disease to blinding eye diseases.
Cell therapies introduce new healthy cells into a patient’s body to replace cells and tissues that have been lost or damaged by age, injury or disease. Center members are pursuing cell therapy methods that use a patient’s own stem cells to address heart damage, neurodegenerative diseases, spinal cord injuries and myriad other conditions. In cases where damage to patients’ cells and tissues is the result of mutated or missing genes, gene therapies can replace, modify or correct faulty genes to treat the underlying causes of disease. These novel therapies can address a range of inherited conditions including cystic fibrosisExcessive scarring within an organ due to disrupted healing. It can lead to organ dysfunction and is associated with conditions like chronic kidney disease, liver cirrhosis and heart failure.fibrosisExcessive scarring within an organ due to disrupted healing. It can lead to organ dysfunction and is associated with conditions like chronic kidney disease, liver cirrhosis and heart failure. and muscular dystrophies.
Our researchers are also innovating approaches that combine gene and cell therapy techniques. Center member Donald Kohn, M.D., has been developing therapies of this kind to treat genetic blood and immune disorders such as sickle cell disease for over 30 years. While advancing several of these therapies through clinical trials, he and his center collaborators are working to enhance and adapt these therapeutic approaches to treat a wider array of diseases.
Our Goals
- Develop and clinically advance gene therapyA technique that uses a gene or gene(s) to prevent, treat or cure a disease or disorder. Most gene therapies work by adding a healthy version of a gene to replace one that is defective or missing into the genome of particular cells. Some of these therapies use viral vectors to deliver genes into target cells.gene therapyA technique that uses a gene or gene(s) to prevent, treat or cure a disease or disorder. Most gene therapies work by adding a healthy version of a gene to replace one that is defective or missing into the genome of particular cells. Some of these therapies use viral vectors to deliver genes into target cells. and gene editingA type of gene therapy that works by delivering genetic material that can directly edit pieces of DNA within a cell. This changes the instructions the DNA encodes for, which ultimately results in an increase or decrease in the production of a certain protein and the restoration of proper cell function.gene editingA type of gene therapy that works by delivering genetic material that can directly edit pieces of DNA within a cell. This changes the instructions the DNA encodes for, which ultimately results in an increase or decrease in the production of a certain protein and the restoration of proper cell function. approaches to treat rare genetic immune disorders including adenosine deaminase–deficient severe combined immunodeficiency, X-linked chronic granulomatous disease and leukocyte adhesion deficiency-I
- Advance stem cell-based therapies that repair brain damage and improve memory function to help patients recover from strokes and dementia
- Develop gene and cell therapies for rare metabolic disorders, which often result in severe intellectual disability or early postnatal death
- Develop and clinically advance gene therapy and gene editing approaches to treat sickle cell disease
- Develop stem cell-engineered, off-the-shelfA type of "universal" cell therapy that can safely be used in any person. These experimental therapies rely on healthy donor cells that — either due to their unique properties or thanks to genetic modification — do not carry the risk of being rejected by patients' immune systems. Because these therapies do not have to be manufactured on a patient-specific basis, they can benefit more patients and reach them in a faster, more cost-effective way. off-the-shelfA type of "universal" cell therapy that can safely be used in any person. These experimental therapies rely on healthy donor cells that — either due to their unique properties or thanks to genetic modification — do not carry the risk of being rejected by patients' immune systems. Because these therapies do not have to be manufactured on a patient-specific basis, they can benefit more patients and reach them in a faster, more cost-effective way. immune cell therapies to treat multiple myeloma, glioblastoma and other blood cancers and solid tumors
- Create stem cell-based therapies that repair damage caused by blinding eye diseases, muscular dystrophies, spinal cord injuries and intestinal conditions
- Develop a gene therapy for cystic fibrosisExcessive scarring within an organ due to disrupted healing. It can lead to organ dysfunction and is associated with conditions like chronic kidney disease, liver cirrhosis and heart failure.fibrosisExcessive scarring within an organ due to disrupted healing. It can lead to organ dysfunction and is associated with conditions like chronic kidney disease, liver cirrhosis and heart failure. that uses nanotechnology to reach disease-affected airway stem cellsCells that have the ability to differentiate into multiple types of cells and make an unlimited number of copies of themselves.stem cellsCells that have the ability to differentiate into multiple types of cells and make an unlimited number of copies of themselves. without injuring patients’ lungs
- Improve affordability of and access to cell and gene therapies by engineering nanotechnology-based solutions for packaging and delivering gene editing reagents to patient cells
Research Highlights
Treating Duchenne muscular dystrophy
Center scientists help gain FDA approval for a gene therapy targeting the progressive muscle-wasting disease Duchenne muscular dystrophy, treating a dozen patients commercially and awaiting others.
Reimagining gene therapy delivery
UCLA researchers work to develop an innovative gene-editing platform that corrects the root cause of cystic fibrosis through an inhalable mist.
Restoring the immune system
Two years after treatment in a UCLA clinical trial, nine children who received an innovative gene therapy for the fatal immune disorder LAD-I are healthy and disease-free.
From transplants to gene therapy
How a transplant surgeon’s connection with patient families inspired his quest for a gene therapy to treat creatine transporter deficiency — a rare genetic disorder that affects children.
Creating lifelong curative therapies
UCLA researchers led by gene therapy pioneer Dr. Donald Kohn are developing a one-time stem cell gene therapy to treat the severe, inherited blood disorder alpha thalassemia.
Gene therapy treats sickle cell disease
After receiving an experimental stem cell gene therapy for sickle cell disease at a UCLA clinical trial, Evie Junior no longer experiences debilitating pain – and 70% of his blood stem cells are free of the disease-causing gene.
Continued patient success
UCLA researchers use a stem cell gene therapy to treat a rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD, in nine patients, six of whom remain in remission without other treatments.
Making bone marrow transplants safer
UCLA researchers work to develop an off-the-shelf cell therapy to reduce the severity of graft-versus-host disease in people receiving donor bone marrow transplants for the treatment of blood cancers such as leukemia and lymphoma.
Enduring results from UCLA-led trial
An experimental gene therapy developed by center researchers successfully treats 59 of 62 children born with ADA-SCID, a rare and deadly inherited disorder that leaves patients without an immune system.
