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Gene Therapy

The introduction, removal or change in genetic material in the cells of a patient to treat an inherited or developed disease.

Gene Addiction

involves inserting a new copy of a gene into the target cells to produce more of a protein.

Gene Correction

can be achieved by modifying part of a gene using recently-developed gene editing technology (e.g. CRISPR/cas9, TALEN or ZFN) to remove repeated or faulty elements of a gene, or to replace a damaged or dysfunctional region of DNA.

Goal of Gene Correction

Produce a protein that functions in a normal manner instead of in a way that contributes to disease.

Gene Sliencing

Inactivating or “knocking out” a mutated gene.

Reprogramming

Involves adding one or more genes to cells of a specific type to change the characteristics of those cells.

Cell elimination

Used to destroy malignant (cancerous) tumor cells, but can also be used to target overgrowth of benign (non-cancerous) tumor cells.

Somatic Cell

targets only the genes in a patient’s body cell, so only the individual who receives the therapy is affected

Germ Line

targets the genes in a patient’s egg or sperm, so changes can be passed on to offspring & not in development

Transgene

It is an experimentally constructed piece of DNA that has integrated into the genome of a recipient organism.

Cell Therapy

The transfer of cells into a patient with the goal of improving a disease

CAR-T cell therapy

Example of Cell Therapy

CAR-T cell therapy

A patient’s T cells, which are a kind of immune cell, are changed in a lab by using a vector to add a gene that changes cells in a way that enables them to recognize and attack cancer cells.

Embryonic stem cells (ESCs)

These are pluripotent stem cells (can give rise to all the types of cell) derived from embryos.

Induced pluripotent stem cells (iPSCs)

A differentiated adult (somatic) cell, such as skin cell is \ reprogrammed to return to a pluripotent state.

Nuclear transfer embryonic stem cells (ntESCs)

These pluripotent cells are produced by transferring the nucleus from an adult cell obtained from the patient to an oocyte (egg cell) obtained from a donor.

Hematopoietic stem cells (HSCs)

are multipotent blood stem cells that give rise to all types of blood cells.

Immune cell therapy

The cells can be removed from the body, isolated from a mixed cell population, modified & then expanded before return to the body.

Mesenchymal, Neural, Epithelial

Other stem cell sources

Stem Cells

Basic cells that have the capacity to create more stem cells & to differentiate into different cell types

Adult stem cells

These stem cells are found in small numbers in most adult tissues, such as bone marrow or fat. Compared with embryonic stem cells, adult stem cells have a more limited ability to give rise to various cells of the body.

Embryonic stem cells

These stem cells come from embryos that are 3 to 5 days old. At this stage, an embryo is called a blastocyst and has about 150 cells.

pluripotent stem cells

they can divide into more stem cells or can become any type of cell in the body. This allows embryonic stem cells to be used to regenerate or repair diseased tissue and organs.

Induced pluripotent cells

are regular skin, fat, liver, nerve, or other cells that scientists have modified to behave like embryonic stem cells

Totipotent

These stem cells can differentiate into all possible cell types. The first few cells that appear as the zygote starts to divide.

Pluripotent

These cells can turn into almost any cell. Cells from the early embryo are pluripotent.

Multipotent

These cells can be differentiate into a closely related family of cells. Adult hematopoietic stem cells, for example, can become red & white blood cells or platelets.

Dolly the sheep

the poster child of modern biotechnology. It was the first mammal to have been successfully cloned from an adult cell.

Administrative Order No. 2013-0012

prohibits and restricts the creation, importation, promotion, marketing and use of stem cell therapies from embryonic, aborted fetal, and genetically altered, animal and plant stem cells.