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Glossary

Home > Gene & Cell Therapy 101 > Glossary

Use the A-Z index below to find words that begin with that letter.

  • A

    • Adaptive immunity

      The part of the immune system that builds up responses to antigens or foreign invaders (like viruses or bacteria) as we are exposed to disease or receive a vaccination. This response can become stronger and more specialized over time or with repeated exposure. 

    • Adeno associated viral (AAV) vector

      A type of virus that does not make people feel sick and is used to deliver genetic material into cells, such as DNA or RNA.

    • Adenovirus

      A type of virus used to deliver small to large DNA packages into cells. Adenoviruses are highly immunogenic (meaning able to produce an immune response) and therefore mostly used for cancer applications. 

    • Adverse event

      Any health problem ranging from minor to serious that happens after receiving a vaccine, treatment, or therapy.  

    • Allele

      Two, or more, sets of genetic sequence present at a particular chromosomal location (locus). One allele is inherited from each parent, and the pair determines the genotype of a given gene.   

    • Allogeneic

      Cells used from someone other than the patient during treatment. A helpful tip to remember this is that "allo" means other. 

    • Amino acids

      The basic building blocks of all proteins, around 20 amino acids make up all animal and plant proteins. 

    • Antibody

      Protein produced by the body to fight infections and foreign antigens.

    • Antigens

      A piece of a foreign invader that the immune system recognizes as not being part of itself, such as the protein surface of a virus.  

    • Antisense oligonucleotide (ASO) therapies

      Utilize ASOs to interact with mRNA in order to inhibit or alter protein function.  

    • Antisense oligonucleotides (ASOs)

      Synthetic (made in a lab) single strand of genetic material that targets mRNA from a specific gene to either increase to decrease production of the protein encoded by the mRNA. 

    • Aptamer

      Synthetic, single-stranded DNA or RNA that can bind a specific target and can sometimes serve as the chemical equivalent of an antibody. 

    • Aptamer therapies

      Utilizes RNA aptamers that can tightly bind to proteins on the outside of the cell to provide unique therapeutic advantages.

    • Autologous

      Cells from an individual to be used during a possible cell therapy on that same individual. Auto means "self," so this may also be considered being a "self-donor."  

    • Autosomal dominant

      A pattern of inheriting a genetic change that is located on one of the non-sex, numbered chromosomes (autosomal). A single copy of the disease-associated gene variant from one parent is enough to pass along a disease. 

    • Autosomal recessive

      A pattern of inheriting a genetic change where two copies of a disease associated gene variant (one on each allele) are needed to pass along a disease. 

    • Autosome

      One of the numbered chromosomes; humans have 22 pairs that are numbered 1 to 22. These do not include the sex chromosomes (X or Y).  

  • B

    • Biologics License Application (BLA)

      Document submitted to the Food and Drug Administration (FDA) if the results of the final phase of the clinical trial show the treatment to be safe and effective, specific to gene/cell therapies, vaccines, and antibody therapies. 

  • C

    • Capsid

      The shell of a virus that is used as the package to deliver the genetic material to cells. 

    • Carrier screen

      A genetic test to tell if a person is carrying a copy of a gene variant known to cause disease. The person (carrier) may not be affected but could pass it to their children who could inherit the trait. 

    • Cell

      The basic unit of every living thing.

    • Cell therapy

      The transfer of a specific cell type into a person to treat or prevent a disease. 

    • Chromosome

      Long strings of DNA tightly wound to form an “X” shape. Human cells contain 23 pairs of chromosomes (46 total), with the mother and the father each contributing one chromosome to each pair in their children. 

    • Clinical trial

      A required part of the research process that aims to understand the way a drug or treatment will interact with the human body and whether it is safe and effective. 

  • D

    • De novo mutations

      A new change in a person’s DNA that causes a genetic disorder that the parents do not have and there is no prior family history. Also known as a new mutation, or new gene variant.  

    • DNA (deoxyribonucleic acid)

      Two strands of connected molecules that store genetic information so the cell can continue building and regulating proteins. It is stored in the form of chromosomes within the nucleus. 

    • DNA therapy

      The use of DNA that codes for the production of a specific RNA or protein to treat a disorder. 

  • E

    • Eligibility criteria

      For every clinical trial there are set guidelines or characteristics for who can and cannot participate in the study, also called inclusion and exclusion criteria. 

    • Emergency Use Authorization

      Order given by the FDA during a public health emergency, to provide access to medical products that may be used when there are no adequate, approved, and available

    • Enzymes

      A type of protein that plays an important role in the chemical reactions that modify molecules (DNA, RNA, other proteins or lipids).  

    • Ex vivo

      Means outside the body. An ex-vivo gene therapy removes the person’s own cells, delivers the new genetic material to these cells, then the modified cells are returned to the body. This is also known as gene modified cell therapy.  

    • Expanded access

      (also known as compassionate use) The pathway by which the FDA allows the use of an investigational therapy (drug, biologic, or device) outside of a clinical trial for patients with a serious or immediately life-threatening disease or condition. This is prior to approval of a treatment and not part of a clinical trial.  

  • F

    • FDA

      United Stated Food and Drug Administration. American agency that regulates the approval and use of items such as food, drugs, medical devices, vaccines, and biologics. 

  • G

    • Gene

      Specific sections of DNA that encode sets of instructions for making and controlling proteins.  

    • Gene activation

      A gene editing approach that activates a previously silent protein. 

    • Gene addition

      A type of gene therapy that adds a working gene that has the instructions for cells to make more of the specific protein needed.  

    • Gene editing

      A type of gene therapy that corrects pieces of DNA permanently by changing or deleting the information within the affected individual’s gene.  

    • Gene integration

      A gene editing approach that inserts the correct DNA sequence into one’s genome so that it can produce functional proteins. 

    • Gene knock out

      A gene editing approach that can be used to silence or “knock out'' certain genes in a cell. This approach alters or removes the DNA sequence entirely. 

    • Gene modified cell therapy

      Also known as ex vivo gene therapy. See ex vivo definition.  

    • Gene silencing

      A type of gene therapy where the delivered genetic material prevents or inhibits the activity of a gene that is already present in the cell. 

    • Gene therapy

      The use of genetic material to treat or prevent disease.  

    • Gene variant

      Also known as a gene mutation, it is a change to the DNA within our genes that can alter the instructions for how proteins are built and work, which can lead to a disease or disorder. 

    • Genetic material

      Deoxyribonucleic acids (DNA) and ribonucleic acids (RNA).

    • Genetic testing

      A variety of tests that look for changes in DNA that can cause human disease. 

  • H

    • Hematopoietic stem cells (HSCs

      Also known as blood forming stem cells, they are versatile cells that can turn into any type of blood cell the body needs and can be retrieved from the peripheral blood, bone marrow, or umbilical cord blood. 

  • I

    • Immunosuppressed

      Having a weakened immune response against pathogens (i.e. virus) leading to an increased risk of infection. It can be temporary or permanent and caused by disease or as an effect of certain drugs. 

    • In vivo

      Means inside the body. An in vivo gene therapy delivers genetic material directly into the person, such as through an injection. 

    • Informed consent

      A series of discussions that will take place before an eligible participant can volunteer for a clinical trial. The researcher will lead discussions to ensure the participant or caregiver understands the details of the trial, what to expect before, during, and after, and what their rights are during the trial. This process continues throughout the trial and requires the participant to sign a document if choosing to be a part of the research study. 

    • Innate immunity

      The first immune system response against a pathogen (i.e. virus) that may attack or destroy a vector before it can deliver its therapeutic payload, lessening or stopping its effectiveness.  

    • Investigational new drug (IND) application

      The way preclinical study data and clinical trial design are submitted for review to a government regulatory agency, such as the FDA.  

  • L

    • Lentivirus

      A type of virus from the retrovirus family that is most commonly used to deliver genetic material in ex-vivo gene therapy. Many of these vectors used today were originally derived from the human immunodeficiency virus type 1 (HIV). HIV is not active in lentivirus vectors. 

    • Lipid nanoparticle

      A protective shell made of fat, or lipids, to carry various payloads to the cells.

  • M

    • Messenger RNA (mRNA)

      A middle message (between DNA and protein) made up of genetic material within the body that contains genetic instructions for cells to make proteins.

    • MicroRNA (miRNA)

      A small, non-protein coding RNA that targets mRNA to regulate expression of genes. 

    • miRNA therapies

      A therapy that carries miRNA encoding sequences (naturally occurring or artificial), or oligonucleotide-based inhibitors (anti-miRs).

    • mRNA therapy

      Introduction of mRNA as a vaccine or therapeutic agent to increase the production of a protein or peptide. 

    • mRNA vaccine

      A vaccine that delivers synthetic (fake) mRNA molecules into cells and instructs them to make antigens to activate an immune response. 

    • Mutation

      A gene variant (a change in DNA) that may or may not cause human disease.

  • N

    • Natural history studies

      Studies that collect information about how a disease progresses in the absence of treatment, often including various functional testing, surveys, and sample donation like blood, skin, or urine. 

    • Neuron

      (Also called nerve cells) are specialized cells that send messages from the brain to every part of the body and back. These cells are essential for every biological process from basic survival (breathing, heart function, heat regulation) to higher level functions (thinking, emotional regulation, decision-making). 

    • Non-viral vector gene therapy

      A type of gene therapy that does not utilize a virus as the vehicle to carry material to the cell, it is often called synthetic gene therapy. 

    • Nucleic acids

      The four building blocks of genetic material that make up DNA and RNA.

    • Nucleus

      The part of a cell that holds the DNA.

  • O

    • Off-target effect

      Occurs when tissues or cells other than the intended target may be affected after administration of a gene therapy.  

  • P

    • Plasmids

      Small, double-stranded circular DNA molecules separate from the cell’s DNA found in the chromosomes. Naturally, these are most commonly found in bacteria. They are used as a tool in gene therapy to introduce genetic material into cells.  

    • Pre-clinical studies

      An early stage of research that tests the safety and effectiveness of a treatment in animal or cell-based models before a human clinical trial can take place. 

    • Proteins

      Molecules made up of amino acids required for the structure, function, and regulation of the body’s tissues and organs. DNA codes for all proteins in the body. 

  • R

    • Retrovirus

      A type of virus that uses RNA to carry its genetic material which is then converted into DNA in the host cell (the cell that is infected by the virus).  

    • RNA (ribonucleic acid)

      A copy of the genetic information within DNA that helps turn the instructions from genes into proteins or prevents those proteins from being made. It is mostly made within the nucleus of the cell, and there are many different types.  

    • RNA therapy

      The use of shorter sequences of genetic material in RNA format to treat or prevent a disease. There are many different types of RNA therapies. 

  • S

    • siRNA therapies

      Utilize siRNA to target expression of a specific gene. 

    • Small interfering RNA (siRNA)

      Double stranded RNA molecules that usually target a specific mRNA to prevent production of unwanted proteins. 

    • Stem cells

      Undefined cells with the potential to become specific cell types, depending on what the body needs. 

    • Suppressor tRNA therapies

      Utilize tRNA to override incorrect mRNA instructions that may cause disease by stopping protein production too early. 

  • T

    • Transfer RNA (tRNA)

      Carry the building blocks of proteins called amino acids, to the ribosome to help make a protein based off the mRNA instructions. 

  • V

    • Vector

      Vehicles to deliver therapeutic (meaning used to treat or prevent disease) genetic material or specific DNA sequences into a cell.

    • Viral vector

      A virus modified to deliver therapeutic genetic material into a cell. Viruses have evolved to be very good at getting into cells, and scientists have learned how to remove the viral genes and use this same ability to treat or prevent disease.  

  • W

    • Whole genome sequencing

      A test that determines the order of the four chemical building blocks, called bases, that make up the DNA molecule. This is a more in-depth form of genetic testing that takes into consideration the whole genome and can also help identify changes causing human disease.  

  • X

    • X-linked

      An inheritance pattern in which the genetic variant is located on the X-chromosome which can be recessive or dominant. X-linked diseases more often affect males as females have two x chromosomes while males only have one. 

  • Y

    • Y-linked

      An inheritance pattern in which the genetic variant is located on the Y-chromosome. Only males have a Y chromosome, so a variant can only be passed from father to son.  

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