Alternatively, once the down stream targets of Rett's silencing effect have been determined, phamra companies have several tools in their arsenal to treat the disease,
from small molecule drugs to RNAi.
Not exact matches
The pH during one of the reactions was a bit too low, causing the
drug to extrude a single
small molecule of propionic acid, which transformed it
from a powerful narcotic into a poison that laid waste to the brain's dopamine receptors.
Unpublished results
from the researchers hint that significantly fewer anomalies are seen in iPS cells created via virus - free reprogramming strategies, such as ones that use proteins or
small -
molecule drugs.
However, this security system is so effective at protecting the brain that it prevents many life - saving
drugs — all but some
small molecules —
from being able to treat cancer and other diseases of the brain.
The researchers behind the new study,
from Winston - Salem State University in the US and Universiti Malaysia Sarawak in Malaysia, have found that adding similar, but
smaller polycationic
molecules onto a new kind of material called carbon nanodots makes them even better at killing
drug - resistant bacteria.
Scientists already have a
drug that can curb Brd4's actions — a
small molecule called JQ1 that prevents Brd4
from binding to chromatin.
The new
drug, a
small molecule called anle138b, blocks these pores
from moving ions in and out of nerve cells.
In the early 2000s, Danishefsky — who is best known for synthesizing Taxol (paclitaxel), a
small -
molecule cancer
drug derived
from the Pacific yew tree — set his sights on synthesizing EPO that would be identical to that in the body.
A typical
small -
molecule drug derives its activity
from such functional groups, which are bound to a relatively simple backbone structure consisting chiefly of carbon atoms.
One effective approach is to deliver
drugs made
from small molecules of ribonucleic acid, or RNA, which are used to inhibit gene expression.
They collected gene expression data
from six genetically diverse breast cancer cell lines, which were each exposed to 109
small -
molecule drugs at multiple dosages and time points.
The consolidation of these efforts in an independent initiative will allow faculty
from the CMI to lead a focused investigation in
small molecule drug discovery for multiple therapeutic applications.
(That's different
from manufacturing a
small -
molecule generic, which will have the exact chemical structure of the brand - name
drug.)
Lipitor and Plavix are
small -
molecule drugs, a class of treatment that includes everything
from aspirin to many of today's most sophisticated
drugs.
Not a light read, this tome is bursting with information about monoclonal antibodies,
from the history of how these therapies were first developed to new approaches such as conjugate therapy, which combines antibodies with
small -
molecule drugs.
«We have provided evidence, in collaboration with Erich Wanker
from Berlin, that such activation can be achieved, in principle, with an experimental
small molecule drug [called] geldanamycin.
Scientists can screen collections of
small organic
molecules — the type of
molecules needed for pill - based
drugs — to find any that compete with the peptides by binding tightly to Gα proteins and preventing them
from transmitting signals.
Figure 2: Comparing the patent landscapes for induced pluripotent stem cell (top) and a
small -
molecule drug candidate (MK - 1775, bottom) highlights need for intellectual property consideration in cell therapy DST — search and analysis conducted in Thomson Innovation and Thomson Data Analyzer (TDA) software
from Thomson Reuters; themescape maps produced using TDA.
Abbreviations: Aβ, amyloid β - peptide; AD, Alzheimer's disease; ALS, amyotrophic lateral sclerosis; Ambra1, activating
molecule in Beclin -1-regulated autophagy; AMPK, AMP - activated protein kinase; APP, amyloid precursor protein; AR, androgen receptor; Atg, autophagy - related; AV, autophagic vacuole; Bcl, B - cell lymphoma; BH3, Bcl - 2 homology 3; CaMKKβ, Ca2 + - dependent protein kinase kinase β; CHMP2B, charged multivesicular body protein 2B; CMA, chaperone - mediated autophagy; 2 ′ 5 ′ ddA, 2 ′, 5 ′ - dideoxyadenosine; deptor, DEP - domain containing mTOR - interacting protein; DRPLA, dentatorubral pallidoluysian atrophy; 4E - BP1, translation initiation factor 4E - binding protein - 1; Epac, exchange protein directly activated by cAMP; ER, endoplasmic reticulum; ERK1 / 2, extracellular - signal - regulated kinase 1/2; ESCRT, endosomal sorting complex required for transport; FAD, familial AD; FDA, U.S. Food and
Drug Administration; FIP200, focal adhesion kinase family - interacting protein of 200 kDa; FoxO3, forkhead box O3; FTD, frontotemporal dementia; FTD3, FTD linked to chromosome 3; GAP, GTPase - activating protein; GR, guanidine retinoid; GSK3, glycogen synthase kinase 3; HD, Huntington's disease; hiPSC, human induced pluripotent stem cell; hVps, mammalian vacuolar protein sorting homologue; IKK, inhibitor of nuclear factor κB kinase; IMPase, inositol monophosphatase; IP3R, Ins (1,4,5) P3 receptor; I1R, imidazoline - 1 receptor; JNK1, c - Jun N - terminal kinase 1; LC3, light chain 3; LD, Lafora disease; L - NAME, NG - nitro - L - arginine methyl ester; LRRK2, leucine - rich repeat kinase 2; MIPS, myo - inositol -1-phosphate synthase; mLST8, mammalian lethal with SEC13 protein 8; MND, motor neuron disease; mTOR, mammalian target of rapamycin; mTORC, mTOR complex; MVB, multivesicular body; NAC, N - acetylcysteine; NBR1, neighbour of BRCA1 gene 1; NOS, nitric oxide synthase; p70S6K, ribosomal protein S6 kinase - 1; PD, Parkinson's disease; PDK1, phosphoinositide - dependent kinase 1; PE, phosphatidylethanolamine; PI3K, phosphoinositide 3 - kinase; PI3KC1a, class Ia PI3K; PI3KC3, class III PI3K; PI3KK, PI3K - related protein kinase; PINK1, PTEN - induced kinase 1; PKA, protein kinase A; PLC, phospholipase C; polyQ, polyglutamine; PS, presenilin; PTEN, phosphatase and tensin homologue deleted
from chromosome 10; Rag, Ras - related GTP - binding protein; raptor, regulatory - associated protein of mTOR; Rheb, Ras homologue enriched in brain; rictor, rapamycin - insensitive companion of mTOR; SBMA, spinobulbar muscular atrophy; SCA, spinocerebellar ataxia; SLC, solute carrier; SMER,
small -
molecule enhancer of rapamycin; SMIR,
small -
molecule inhibitor of rapamycin; SNARE, N - ethylmaleimide - sensitive factor - attachment protein receptor; SOD1, copper / zinc superoxide dismutase 1; TFEB, transcription factor EB; TOR, target of rapamycin; TSC, tuberous sclerosis complex; ULK1, UNC -51-like kinase 1; UVRAG, UV irradiation resistance - associated gene; VAMP, vesicle - associated membrane protein; v - ATPase, vacuolar H + - ATPase; Vps, vacuolar protein sorting
The targets can vary
from things that are very
small, like a
drug molecule to something much larger, like bacteria or viruses.
Its pharmaceutical products include chemically - synthesized
drugs or
small molecules and an increasing portion of products produced
from biological processes called biologics.
These large
molecule drugs differ
from traditional pharmaceuticals in that they are usually injected, while
small molecule pharmaceuticals are taken in tablet form.