Glossary



AIDS

 


Acquired immune deficiency syndrome or acquired immunodeficiency syndrome (AIDS) is a set of symptoms and infections resulting from the damage to the human immune system caused by the human immunodeficiency virus (HIV).  This condition progressively reduces the effectiveness of the immune system and leaves individuals susceptible to opportunistic infections and tumors.


Antibody

 


A Y-shaped protein on the surface of B cells that is secreted into the blood or lymph in response to the presence of an antigen. Each antibody binds to a specific antigen to induce cell apoptosis (cell death).


Antigen

 


A protein on the surface of a cell capable of inducing a specific immune response.


Apoptosis

 


The process leading to controlled cellular self-destruction (cell "suicide").


AZT

 


Azidothymidine (AZT) is a nucleoside analog reverse transcriptase inhibitor, a type of antiretroviral drug. It was the first approved treatment for HIV. It is also sold under the names Retrovir. Zidovudine was the first drug approved for the treatment of AIDS and HIV infection. It is also the drug that can be used to delete cells that express the TMPK gene.


Cancer

 


A family of diseases in which cells grow and spread uncontrollably throughout the body disrupting the balance between new cell growth and old cell death.


Chronic Lymphocytic Leukemia

 


Chronic lymphocytic leukemia is a type of cancer of the white blood cells. CLL is an abnormal neoplastic proliferation of B cells and they accumulate to large numbers in the bone marrow and blood.


DNA (deoxyribonucleic acid)

 


The building block of living organisms that carries the genetic information in a cell and is able to self replicate and synthesize RNA.


Erythropoietin

 

 


Erythropoietin is a glycoprotein hormone that controls erythropoiesis, or red blood cell production. It is produced by the liver and kidney, and is the hormone that regulates the number of red cells in the body.


Ex vivo

 


Outside the living body.


Gene

 


A hereditary unit (comprised of DNA) that carries the instructions for making the thousands of proteins needed for a specific cellular functions. Certain diseases are associated with the absence or malfunction of a specific gene.


Graft vs Host Disease

 


Graft-versus-host disease (GVHD) is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack.


Glioblastoma multiforme

 


Glioblastoma multiforme (GBM) is the most common and most aggressive type of primary brain tumor, accounting for 52% of all primary brain tumor cases and 20% of all intracranial tumors. Despite being the most prevalent form of primary brain tumor, GBMs occur in only 2-3 cases per 100,000 people in Europe and North America.


Hemophila A

 

 


Hemophilia A is a blood clotting disorder caused by a mutation of the factor VIII gene, leading to a deficiency in Factor VIII. It is the most common hemophilia . Inheritance is X-linked recessive; hence, males are affected sexually while females are carriers or very rarely display a mild phenotype . 1 in 5,000 males are affected.


HIV

 

 


Human immunodeficiency virus (HIV) is a lentivirus (a member of the retrovirus family) that can lead to acquired immunodeficiency syndrome (AIDS), a condition in humans in which the immune system begins to fail, leading to life-threatening opportunistic infections.


Immunotherapy

 

 


An approach to the treatment of disease that seeks to stimulate and enhance the body's natural immune system to fight the disease.


In vitro

 


Observable in a test tube.


In vivo

 

 
Inside the living body.


Lentiviral Vector

 


A viral gene delivery system engineered for in vivo delivery of therapeutic genes into both dividing and nondividing cells. Lentiviruses have the capability to insert a significant amount of genetic information directly into the DNA blueprint of the host's cells making the lentivirus one of the most efficient methods of gene delivery.


Metastasis

 

 

 


The process by which cancer spreads from a primary location in the body to other healthy tissues located elsewhere in the body via the lymphatic and circulatory systems.


MGMT

 


Methylguanine DNA methyltransferase is the gene that encodes the DNA repair protein O 6-alkylguanine-DNA alkyltransferase. A mutant form of the protein (P140K) makes cells resistant to the effects of DNA alkylating agents such as Temozolomide (TMZ) in the presence of O 6-benzylguanine (BG), a drug that potentiates the effects of TMZ.


Original Biologics Manufacturer(OBM)

 


A manufacturer of biologic materials that is provided to third parties for sale to the scientific community.


Proteins

 


Complex molecules responsible for specific and unique functions within the body. Examples of proteins include hormones, enzymes, and antibodies.


Therapeutic Index

 


The relative efficiency of one mode of treatment compared to another.


Vaccine

 


A vaccine is a biological preparation which is used to establish or improve immunity to a particular disease.


Vector

 


The vehicle by which genes are transported into cells thereby allowing cellular genetic modification to occur. Viral vectors are viruses rendered incapable of reproducing themselves and non viral vectors are "naked" DNA or lipid coated DNA.


Virus

 


A microorganism that requires a host organism in order to grow and replicate. A replicating virus integrates its genetic information (DNA or RNA) into the host cell "overriding" the host cell's biological mechanism in order to reproduce new virus particles.

 
 
 
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