Forever Fix 1-3 Flashcards
Phase 1 Clinical Trial
An early study of a medical procedure conducted to assess safety
Principal Investigator
The lead scientist who has the main responsibility for designing and conducting the experiment
Stem Cell
Partially differentiated cell that can form progeny with a specific function
SNP
A change to one base pair in the DNA sequence
Gene Therapy
A procedure that inserts normal genes into cells with defective genes to correct the function
Adenovirus
A family of viruses containing double-stranded DNA. They cause a range of human illnesses and are used in gene therapy.
Photoreceptors
Rods and cones in the back of the eye responsible for capturing light and sending signals to the brain
Electroretinogram (ERG)
Test that shows the retinas’ responses to light
Leber Congenital Amaurosis (LCA)
Genetic disease where cells cannot produce functioning retinal pigment epithelium (RPE) to absorb stray light rays and store vitamin A for the eyes.
Adenosine Deaminase Deficiency (ADA)
A lack of adenosine deaminase stops a key biochemical reaction in a way that causes the buildup of a toxin (deoxyadenosine) that in turn poisons healthy T cells.
Adrenoleukodystrophy (ALD)
Disorder that prevents the breakdown of VLCFAs, thus leading to VLCFA build up and difficulty sending and receiving brain signals
Cytokine
Any of a class of immunoregulatory proteins that are secreted by cells especially of the immune system
Dysregulation
Impairment of a physiological regulatory mechanism that governs metabolism, immune response, or an organ
Graft-Versus-Host Disease
Bodily condition that results when T cells from a tissue or organ transplant and especially a bone marrow transplant react immunologically against the recipient’s antigens attacking cells and tissues
Immune Sustem
The bodily system that protects the body from foreign substances, cells, and tissues by producing the immune response that includes especially the thymus, spleen, lymph nodes, special deposits of lymphoid tissue, macrophages, lymphocytes, and antigens
Interleukin
Any of various cytokines of low molecular weight that are produced by lymphocytes, macrophages, and monocytes and that function especially in regulation of the immune system and cell-mediate immunity
In-vitro Fertilization with Preimplantation Genetic Diagnosis (IVF-PGD)
An egg is fertilized in the lab dish and a few cell divisions are allowed to occur, then a cell is plucked from the time, early embryo is tested for family’s mutation
Lentivirus
Any of a genus of retroviruses that cause slowly progressive, often fatal human and animal diseases
Lorenzo’s Oil
A mix of canola, olive, and mustard seed oil containing C18 and C22 fatty acids. These fatty acids sop up the enzyme needed to make the deleterious fatty acids that cause ALD, thus lowering their levels
Peroxisome
A cytoplasmic cell organelle containing enzymes which act in oxidative reactions.
Phenylketonuria (PKU)
Disorder resulting from a missing or nonworking enzyme that converts phenylalanine into tyrosine
Retrovirus
Any of a family of single-stranded ran viruses that produce reverse transcriptase by means of which DNA is produced using their ran as a template and incorporated into the genome of infected cells
Rheumatoid Arthritis (RA)
A usually chronic autoimmune disease that is characterized especially by pain, stiffness, inflammation, swelling, and sometimes destruction of joints
Severe Combined Immune Deficiency X-1 (SCID X-1)
Receptors located on T cells which bind interleukins that trigger beneficial immune responses are either defective or absent, thus the message to respond to infection does not reach T cells
T Cell
Any of several lymphocytes that differentiate in the thymus, possess highly specific cell-surface antigen receptors, and include some that control the initiation or suppression of cell-mediated and humoral immunity
Vector
An organism/medium that transmits a pathogen
Very Long Chain Fatty Acid (VLCFA)
Lengthy strands of deleterious fatty acid that build up in the brain, adrenal glands, skin, and blood
