Forever Fix 1-3

Question 0

Phase 1 Clinical Trial

Answer 0

An early study of a medical procedure conducted to assess safety

Question 1

Principal Investigator

Answer 1

The lead scientist who has the main responsibility for designing and conducting the experiment

Question 2

Stem Cell

Answer 2

Partially differentiated cell that can form progeny with a specific function

Question 3

SNP

Answer 3

A change to one base pair in the DNA sequence

Question 4

Gene Therapy

Answer 4

A procedure that inserts normal genes into cells with defective genes to correct the function

Question 5

Adenovirus

Answer 5

A family of viruses containing double-stranded DNA. They cause a range of human illnesses and are used in gene therapy.

Question 6

Photoreceptors

Answer 6

Rods and cones in the back of the eye responsible for capturing light and sending signals to the brain

Question 7

Electroretinogram (ERG)

Answer 7

Test that shows the retinas’ responses to light

Question 8

Leber Congenital Amaurosis (LCA)

Answer 8

Genetic disease where cells cannot produce functioning retinal pigment epithelium (RPE) to absorb stray light rays and store vitamin A for the eyes.

Question 9

Adenosine Deaminase Deficiency (ADA)

Answer 9

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.

Question 10

Adrenoleukodystrophy (ALD)

Answer 10

Disorder that prevents the breakdown of VLCFAs, thus leading to VLCFA build up and difficulty sending and receiving brain signals

Question 11

Cytokine

Answer 11

Any of a class of immunoregulatory proteins that are secreted by cells especially of the immune system

Question 12

Dysregulation

Answer 12

Impairment of a physiological regulatory mechanism that governs metabolism, immune response, or an organ

Question 13

Graft-Versus-Host Disease

Answer 13

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

Question 14

Immune Sustem

Answer 14

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

Question 15

Interleukin

Answer 15

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

Question 16

In-vitro Fertilization with Preimplantation Genetic Diagnosis (IVF-PGD)

Answer 16

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

Question 17

Lentivirus

Answer 17

Any of a genus of retroviruses that cause slowly progressive, often fatal human and animal diseases

Question 18

Lorenzo’s Oil

Answer 18

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

Question 19

Peroxisome

Answer 19

A cytoplasmic cell organelle containing enzymes which act in oxidative reactions.

Question 20

Phenylketonuria (PKU)

Answer 20

Disorder resulting from a missing or nonworking enzyme that converts phenylalanine into tyrosine

Question 21

Retrovirus

Answer 21

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

Question 22

Rheumatoid Arthritis (RA)

Answer 22

A usually chronic autoimmune disease that is characterized especially by pain, stiffness, inflammation, swelling, and sometimes destruction of joints

Question 23

Severe Combined Immune Deficiency X-1 (SCID X-1)

Answer 23

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

Question 24

T Cell

Answer 24

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

Question 25

Vector

Answer 25

An organism/medium that transmits a pathogen

Question 26

Very Long Chain Fatty Acid (VLCFA)

Answer 26

Lengthy strands of deleterious fatty acid that build up in the brain, adrenal glands, skin, and blood