Glossary of Terms

Adenovirus is a virus that causes respiratory tract and eye infections. Adenoviruses used in gene therapy are altered to carry a specific tumor-fighting gene.

Anti-angiogenesis prevents the growth of new blood vessels. Tumors need their own blood supply to survive and by halting the development of new blood vessels, the tumor’s supply of oxygen and nutrients are cut off, thus destroying the tumor.

Antibody is a specialized immune protein directed at foreign proteins, microorganisms or toxins, to fight disease.

Antigen is a substance that, when introduced into the body, stimulates the production of an antibody. Antigens include toxins, bacteria, foreign blood cells, and the cells of transplanted organs.

Apoptosis is programmed cell death, the body’s normal method of disposing of damaged, unwanted or unneeded cells, if they are recognized as such.

B-cells are lymphocytes that make antibodies that attack bacteria and toxins. B-cells are an essential component of the adaptive immune system.

Biomarkers are biochemical features or facets that can be used to measure the progress of disease or the effects of treatment.

Carcinoma is an invasive malignant tumor.

Cell Therapy is the infusion or transplantation of whole cells into a patient for treatment of an inherited or acquired disease.

Checkpoint inhibitors block immune checkpoints. Most currently in use are monoclonal antibodies including Opdivo & Keytruda, which target PD-1. Certain proteins made by some immune system cells, such as T-cells, and some cancer cells help keep immune responses in check and can keep T-cells from killing cancer cells. When these proteins are blocked, the “brakes” on the immune system are released and T-cells are better able to kill cancer cells.

Chimeric antigen receptor T cells (CAR-Ts) a type of engineered T-cell used to kill tumors. They are equipped with special receptors called chimeric antigen receptors (CARs) that recognize specific proteins on cancer cells and eliminate those cells.

Chromosomes are long strings of genetic material made up of DNA and proteins. The DNA contains the approximately 30,000 to 100,000 genes that make up the human genome. Human cells contain 23 pairs of chromosomes (= 46 total), with mother and father each contributing one chromosome to each pair.

Clinical Translation is the translation of research from animal models to humans.

Clinical Trial is designed to answer specific questions about vaccines, new therapies, or new ways of using known treatments. Clinical trials invite human volunteers to determine whether new drugs or treatments are both safe and effective.

Clinical trials are conducted in four phases:
Phase I tests a new drug or treatment in a small group for safety;
Phase II expands the study to a larger group of people for safety and effectiveness;
Phase III groups are as large as several thousand to confirm results; and
Phase IV defines the drug or treatment risks and benefits for government approval.

CRISPR CRISPR-Cas9 (Clustered regularly interspaced short palindromic repeats) repairs a damaged gene by making edits at a particular point in the gene sequence by cutting out, replacing or adding parts to the DNA sequence.

Dendritic cells are the most effective antigen-presenting cells known. They help the immune system recognize cancer cells. They break down cancer cells into smaller pieces (called antigens), then “present,” these antigens to T-cells. This allows the immune system cells to recognize and attack the cancer cells.

DNA [deoxyribonucleic acid] carries genetic information in the cell and is capable of self-replication and synthesis of RNA. DNA consists of two long chains of nucleotides twisted into a double helix and joined by hydrogen bonds between the complementary bases adenine and thymine or cytosine and guanine. The sequence of nucleotides determines individual hereditary characteristics.

Ex vivo gene therapy is the process when cells from a patient are removed, cultivated in the laboratory, and then injected back into the patient. This is called ex vivo because the cells are studied outside the patient’s body.

Gene is a segment of DNA that codes for a particular protein. Humans have over 30,000 genes that act as a blueprint for making specific enzymes or other proteins for virtually every biomedical reaction and body structure.

Gene therapy is a technique that uses genes to treat or prevent disease such as cancer by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including: Replacing a mutated gene that causes disease with a healthy copy of the gene. Inactivating, or “knocking out,” a mutated gene that is functioning improperly. Introducing a new gene into the body to help fight a disease.

Genome is the sum of all genes that code for a particular organism including humans.

Genomics is the study of all of the nucleotide sequences, including structural genes, regulatory sequences and non-coding DNA segments, in the chromosome of an organism.

Hematopoietic cells are the various types of cells that comprise blood, including red blood cells, platelets, macrophages, basophils, eosinophils, neutrophils, and white blood cells (B and T lymphocytes).

Immune Checkpoints molecular switches that tend to decrease immune response.

Immunotherapy, immune-mediated gene therapy, stimulates or restores the ability of the immune system to fight infection and disease. Research hopes to harness immune mechanisms against tumor cells, by using the patient’s own immune system or by transferring of antibodies or T-cells from an outside source.

In vivo gene therapy is when a gene is transferred to cells inside the patient’s body by a vector carrying the therapeutic genetic material. This form of gene therapy is in vivo because the gene is transferred to cells inside the patient’s body.

Leukemia is cancer of the blood. Lymphoma is a cancer of the lymph nodes.

Lymphocytes are small white blood cells that play a large role in defending the body against disease. Lymphocytes are responsible for immune responses via B and T cells. Lymphocytes also secrete products (lymphokines) that modulate the functional activities of many other types of cells and lymphocytes are often present at sites of chronic inflammation.

Metastatic is a description of cancer that has spread from the primary site to nearby or distant structures or organs. When cancer spreads, it is still named after the primary site where it started– for example, the prostate.

Monoclonal Antibody is a group of cells produced from a single cell by repeated cellular replication.

Nanotechnology offers the ability to study and interact with normal and cancer cells in real time, and during the earliest stages of the cancer process using particles 50-200 nm in diameter. These particles allow delivery to overcome barriers for entering cells.

Oncogene is a gene that regulates growth under normal conditions, but sometimes develops a mutation that may contribute to the transformation of normal cells into cancer cells.

Oncogenesis is the process in which a damaged cell continues dividing without restrictions, developing into cancer.

P53 is a multifunctional tumor suppressor protein. This encoding gene is the most commonly mutated gene in tumor cells.

Preclinical Trial refers to the testing of experimental drugs in the test tube or in animals – the testing that occurs before trials in humans are permitted.

Retrovirus is a type of virus that contains RNA whereby a “reverse” transcriptase makes DNA from the RNA template when it is inserted into a cell. Retroviruses cause many diseases, including some cancers and AIDS.

RNA [Ribonucleic acid] is a molecule that is chemically similar to DNA and carries the same code. DNA is biochemically transcribed by enzymes; the transcription product is RNA. This RNA is read, in turn, by the cell’s biochemical machinery to make protein.

Sarcoma is a malignant tumor arising from connective tissue cells.

Somatic Therapy – Involves the manipulation of gene expression in cells that will correct genetic defects of the patient but is not inherited by the next generation. Somatic cells include all the non-reproductive cells in the human body.

Stromal cells are non-hematopoietic stem cells derived from blood organs such as bone marrow, which are capable of supporting the growth of blood cells.

T-cells belong to a group of white blood cells known as lymphocytes and play a central role in cell-mediated immunity. They mature in the thymus gland and attack body cells when they have been taken over by viruses.

Targeted Gene Therapy involves replacement of an abnormal gene with a properly functioning gene. The normal gene is often delivered using a vector.

Translational is the translation of research from animal models to human clinical trials.

Tumor-Specific Replicating Viruses have been modified so they selectively replicate in the tumor and not in normal tissue.

Tumor suppressor gene is a protective gene that normally limits the growth of tumors.

Vector is a gene therapy delivery vehicle or carrier that encapsulates therapeutic genes for delivery to cells.

Viral vectors [disabled, not live virus] transfer tumor antigens (proteins found on a tumor cell) which should stimulate an anti-tumor immune response in the body. Viral vectors may also be used to carry genes that can differentiate cancer cells back to normal cells. Vector development seeks to simulate vectors to combat cancer.