Educational Links

Cell and Gene Therapy

Traditional approaches to treating patients using medicines and surgery have improved the lives of people throughout the world but there are many diseases, perhaps even most of them, for which these traditional approaches modulate the course of the disease but do not provide a cure. This shortcoming is particularly apparent for treating monogenic diseases, diseases caused by mutations in single genes that a person is born with. It is estimated that there are greater than 6,000 such diseases affecting over 350 million people worldwide. Cells and genetically engineered cells have fundamentally different properties than medicines and surgery—they are “living drugs” that can heal and replace damaged tissues or diseased organs. These properties mean that they have the potential to provide curative therapies to a range of diseases that currently have no cure.

Sickle Cell Disease

Group of inherited (genetic mutation) disorders that affect hemoglobin, the molecule in red blood cells that carries oxygen throughout the body. We are involved in a study that will lead to a clinical trial to treat patients with sickle cell disease.

CRISP-R:

Clustered Regularly Interspaced Short Palindromic Repeats

NPR.org

Gene-Edited 'Supercells' Make Progress In Fight Against Sickle Cell Disease

Researchers edited the DNA in bone marrow cells taken from a Mississippi woman with sickle cell disease to produce a treatment that could alleviate the excruciating effects of her inherited illness.

Stanford School of Engineering

Could gene editing enable us to reverse some of the ravages of aging?

Stanford Engineering Digital Magazine Article

Hematological Disorders or Cancers

Blood cancers are hematological disorders that affect the cells that make up blood, bone marrow, and lymph. Examples of this type of cancer are Leukemia and B-cell Lymphomas. More information of Hematological disorders, cancers and treatment options can be found here. Treatment for hematological cancers include CAR-T therapy.

Hematology Program

CAR-T Cell Therapy Brings New Hope to Cancer Patients

Treating many forms of leukemia, and blood disorders such as anemia and multiple myeloma, our dedicated physicians provide expert, multispecialty care.

Behind the Scenes of a Radical New Cancer Cure

The Scientific American blog discusses the history and innovation of CAR-T.

News Center

Stanford researchers program cancer-fighting cells to resist exhaustion, attack solid tumors in mice

CAR-T cells are remarkably effective against blood cancers, but their effect can be transient as the cells become exhausted. Stanford researchers found a way to keep the cells effective in mice with human tumors.

Epidermolysis Bulosa (EB)

Is a rare skin disease that is caused by a mutation in the genes that code for collagen proteins (collagen is the protein that holds your skin together and on you). This disease is on a spectrum with severity of symptoms related to what genes are affected. Recessive dystropic (RD) EB is the most severe form. Babies born this form of EB have extraordinarily fragile skin (“butterfly skin”) and wounds that cannot heal. Our team is developing and manufacturing skin grafts to treat these patients.

Stanford Medicine

The butterfly effect

Skin too fragile to touch

Genetics Home Reference

Dystrophic epidermolysis bullosa

Researchers classify dystrophic epidermolysis bullosa into three major types. Although the types differ in severity, their features overlap significantly and they are caused by mutations in the same gene.

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