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Aromatic L-amino acid decarboxylase, AADC, is the enzyme responsible for converting L-DOPA into Dopamine. Within the brain, AADC is most abundant in a region called the substantia nigra, which contains the neurons that degenerate in Parkinson's disease. This loss of AADC activity through depletion of host cells is believed to play a major role in the progressive loss of response to the drug, L-DOPA, as the disease advances. This phenomenon forms the basis of an enzyme replacement strategy where the the AADC gene is introduced into the striatum to restore L-DOPA responsiveness. AADC is also found in peripheral tissues (liver, kidney, adipose, heart, adrenal gland and keratinocytes). (AADC)


Adeno-associated virus (AAV) is a small virus which infects humans and some other primate species without causing disease. AAV can infect both dividing and non-dividing cells and only rarely incorporates its genome into that of the host cell. The DNA that comprises the viral genome can be largely replaced so that the engineered virus will express a therapeutic protein, without affecting the ability of the virus to interact with target cells. Engineered AAV is also known as a viral vector, a way to use components of a virus to produce therapeutic proteins at specific regions within the body. (AAV)

Alzheimer's Disease

Add definition (AD)


The growth factor brain-derived neurotrophic factor (BDNF) and its receptor tropomyosin-related kinase receptor type B (TRKB) are produced and trafficked in the adult brain. BDNF becomes deficient in the entorhinal cortex and hippocampus in Alzheimer's disease. (BDNF)


A cannula (plural: cannulae) is a flexible tube which can be inserted into the body either to withdraw fluid or to deliver medication. The most widely known cannulae are used for intravenous delivery of fluids or medication. The cannulae used for convection-enhanced delivery are engineered of different materials and are designed much more precisely. (Cannula)


Complementary DNA (cDNA) is DNA that encodes a protein and is synthesized from a mature messenger RNA template in a reaction catalyzed by the enzyme reverse transcriptase. cDNA can be engineered into viral vectors like AAV to enable the production of the protein encoded by the cDNA sequence wherever the vector is delivered. (cDNA)

Pressurized Infusions

Previously called convection-enhanced delivery (CED), pressurized infusions allow for the safe, targeted, homogeneous delivery of agents into small and large tissue volumes at rates multiple orders of magnitude larger than diffusion-driven processes (for large molecules). This is done so in a manner that bypasses the blood-nervous system barrier. Via step-designed cannulae, a high interstitial pressure is generated beyond the cranium into the brain in the porous Virchow-Robbins space that lies between cells around the outside of blood vessels.


Dopamine is a hormone and neurotransmitter occurring in a wide variety of animals, including both vertebrates and invertebrates. It is important for the control of motor function and neuron-to-neuron signaling. (Dopamine)


Dystonia (or dyskinesia) is a neurological movement disorder in which sustained muscle contractions cause twisting and repetitive movements or abnormal postures. (Dyskinesia)


FMT stands for fluoro-metatyrosine, a synthetic substrate for AADC. The enzyme converts FMT mainly to 6-fluoro-hydroxyphenylacetic acid, and this metabolite remains trapped within the tissue where AADC activity is present. Positron emission tomography (PET) is a nuclear medicine imaging technique that produces a three-dimensional image or map of functional processes in the body. The approximate level of AADC activity can be gauged by the FMT-PET signal recorded by a PET scanner. (PET)

Gadolinium and Gadoteridol

Gadolinium is an element that is opaque to MRI techniques. It can therefore act as a marker on an MRI to identify where it has been deposited. In the Bankiewicz lab, we deliver a version of gadolinium called gadoteridol (known commercially as Prohance) along with the drug being tested to reveal where the drug has reached within the brain. (Gadolinium)

Gene Therapy

Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease. It is used to provide functioning copies of genes in diseases in which the gene functions incorrectly or does not function at all. (Gene therapy)

Glia-Derived Growth Factor (GDNF)

Glial cell line-Derived Neurotrophic Factor (GDNF) is a small protein that potently promotes the survival of many types of neurons, most especially dopaminergic neurons. The death of these neurons is the underlying cause of Parkinson’s disease. (GDNF)

Glioblastoma Multiforme

Glioblastoma multiforme (GBM), also known as grade 4 astrocytoma, is the most common and aggressive type of primary brain tumor, accounting for 52% of all primary brain tumor cases and 20% of all intracranial tumors. Despite standard treatment – including surgical resection, radiation therapy and chemotherapy - median survival time for GBM has remained short for many years: approximately 15 months. (Glioblastoma multiforme)

Huntington's Disease

Huntington's disease (HD), also known as Huntington disease and previously as Huntington's chorea and chorea maior, is a rare inherited neurological disorder affecting up to almost 8 people per 100,000. For unknown reasons, neurons in the brain begin to die off, leading to characteristic movement and cognitive disorders, including dementia. (Huntington's disease)


Interleukin-10 (IL-10 or IL10), also known as human cytokine synthesis inhibitory factor (CSIF), is a cytokine that counteracts the effects of many inflammatory pathways. It also has varied functions in immune system regulation. (IL-10)

L-DOPA (Sinemet®)

Levodopa is a chemical precursor in the synthesis of dopamine, as well as epinephrine and norepinephrine. In the treatment of Parkinson’s disease, it is used as a prodrug to increase dopamine levels for the treatment of Parkinson's disease, since it is able to cross the blood-brain barrier whereas dopamine itself cannot. Once levodopa has entered the central nervous system (CNS), it is converted to dopamine by the enzyme AADC. Sinemet® is a medication containing L-DOPA that is used to treat Parkinson's disease. (L-DOPA)

Liposomal Gadoteridol

The drug, gadoteridol, is a chelated form of the element gadolinium. By incorporating gadoteridol into small lipid globules called liposomes, we have created a reagent that can be co-infused into brain tissue with AAV, drugs, or nanoparticles to track their delivery and distribution within the brain. This material is visible in magnetic resonance imaging (MRI) movies on our website as a white signal. (Gadoteridol)


A liposome is a spherical vesicle composed of a bilayer membrane. Drugs can be incorporated within liposomes to improve their delivery characteristics within the body. (Liposome)


MPTP (1-methyl 4-phenyl 1,2,3,6-tetrahydropyridine) is a chemical that is related to opioid analgesics. MPTP causes Parkinsonian side-effects. It is a possible side product in the synthesis of MPPP, an illicit morphine-like drug, and hence some users of MPPP can develop chemically-induced Parkinsonian symptoms. (MPTP)


Magnetic resonance imaging (MRI), formerly referred to as magnetic resonance tomography (MRT) and, in scientific circles, nuclear magnetic resonance imaging (NMRI) or NMR zeugmatography imaging, is a non-invasive method used to render images of structures within the body. (MRI)


Neurodegeneration is progressive loss of structure or function of neurons, including death of neurons. (Neurodegeneration)

Niemann-Pick Disease

Niemann-Pick disease is a fatal inherited condition involving lipid metabolism (the breakdown and use of fats and cholesterol in the body) in which harmful amounts of lipids accumulate in the spleen, liver, lungs, bone marrow, and brain. Type A disease is caused by a mutation in the acid sphingomyelinase gene resulting in a complete deficiency of the gene. Most cases of Type A disease are fatal by 18 months of age. (Niemann-Pick disease)

Nigra (substantia nigra)

The substantia nigra (Latin for "black substance") or locus niger is a heterogeneous portion of the midbrain. It is a major element of the basal ganglia system. Cells in the substantia nigra produce dopamine, and also contain melanin, a dark pigment that gives the substantia nigra its name. (Substantia nigra)

Parkinson's Disease

Parkinson's disease (also known as Parkinson disease or PD) is a degenerative disorder of the central nervous system that often impairs the sufferer's motor skills and speech. The symptoms are due to the degeneration and loss of dopamine-producing neurons in the substantia nigra. (Parkinson's disease)


The putamen is a structure in the middle of the brain, which together with the caudate nucleus forms the dorsal striatum. As a principal structure in the basal ganglia of the brain, it is important in motor function. (Putamen)

Reflux-Resistant Cannula

A cannula designed by our lab that allows for an even, spherical distribution of infused material. This is achieved by putting a tapered step near the cannula tip that prevents reflux of infusate along the cannula tract.

Sphinogmyelinase (hASM)

Acid Sphingomyelinase is an enzyme that resides in an organelle called the lysosome. It is responsible for breakdown of sphingomyelin, a constituent of many cell membranes. Absence of the enzyme causes a rare genetic disease called Niemann-Pick disease.


The striatum is a subcortical part of the telencephalon, part of the forebrain. It is the major input station of the basal ganglia system. Anatomically, the striatum is comprised of the caudate nucleus and the putamen. (Striatum)

Viral Vector

Viral vectors are a tool commonly used by molecular biologists to deliver genetic material into cells. Viral DNA sequences that express proteins responsible for the virus’ pathogenic activity are removed and replaced with DNA that can regulate and express beneficial genes. The proteins on the viral surface that the virus uses to target specific cells are unaffected by this replacement. (Viral vector)