Sentences with phrase «with small molecule drugs»

Efforts to inhibit this enzyme, called β - secretase 1 (BACE1), with small molecule drugs have met with limited success so far, says Ryan Watts, a neurobiologist at the biotech company Genentech in South San Francisco, California, and one of the leaders of the new research.
By getting inside the cell and getting the body to produce intracellular proteins, we have the potential to address many diseases that today aren't druggable with small molecule drugs or biologics.
Because LINGO1 has be shown to have a negative impact on neuronal development and survival, inibiting it with a small molecule drug may provide an effective approach for developing a treatment for the severe forms of essential tremor.

Not exact matches

Shukla and colleagues discovered that a small drug molecule called BX795, which is sold to labs for use in experiments, helped clear HSV - 1 infection in cultured human corneal cells, in donated human corneas, and in the corneas of mice infected with HSV - 1.
3BP, part of a class of compounds known as small molecule drugs, was first studied as an anti-cancer agent more than a decade ago at Johns Hopkins by biochemists Young Hee Ko and Peter Pedersen, together with radiologist Jeff Geschwind.
If indole allows pathogenic bacteria to withstand antibiotics, it may be possible to thwart drug resistance by blocking indole signaling with small molecules, Collins says.
Initial results were discouraging: attempts to simply replace the protein therapies with small - molecule drugs that hit the same targets largely failed, says Saeed Fatenejad, a rheumatologist at Pfizer who is responsible for clinical development of tasocitinib.
The researchers created a new gel, based on small molecules which self - assemble into nanofibers which could interact with a variety of anti-inflammatory, painkiller drugs, including iburofen and naproxen.
The approach should work with other small - molecule drugs and may lend itself to multiplexing, he notes, so numerous drugs could be measured at the same time.
By studying the regulation of the clock proteins called Period (PER) and Cryptochrome (CRY)-- proteins that are thought to be involved with metabolism — St. John and Doyle were able to model the mechanisms of two small - molecule drugs — Longdaysin and KL0001 — that regulate the expression of the clock proteins.
Coincidentally, co-author Lawrence Shapiro, PhD, at Columbia, was building a crystal structure of cadherin - 11 and is now working with Byers and Brenner to show how the molecule binds to Celebrex and other small molecule drug cadherin - 11 inhibitors.
«One of our experiments showed that bypassing Lin28a and directly activating mitochondrial metabolism with a small - molecule compound also had the effect of enhancing wound healing, suggesting that it could be possible to use drugs to promote tissue repair in humans.»
The researchers tested two anti-CK2 drugs for their ability to stimulate the production of new brown fat in mice: a new small - molecule CK2 - blocker called silmitasertib (CX - 4945), which is already in clinical trials as a cancer therapeutic; and a more precise next - generation antisense oligonucleotide (ASO) drug developed in collaboration with Isis Pharmaceuticals, which eliminates CK2 by blocking the RNA instructions cells use to produce it.
Small molecule drugs were used to inhibit the function of a cellular pathway associated with protein synthesis.
Most do their job well, but they come with a major drawback: «Most drugs in use right now are small molecules, which are very tiny and nonspecific.
Most drugs on the market today are small molecules, active organic compounds with a structure and weight so minute that they can be ingested and pass more easily through cell membranes.
Blocking this process with a small - molecule drug prevented the characteristic brain plaques and memory loss in a mouse model of the disease.
The basic problem the team faced with these and other vaccines they've developed is that the opioid drugs are small molecules, which are not recognized by the immune system.
Small peptides have the benefits of small molecule drugs, like aspirin, and large antibody therapies, like rituximab, with fewer drawbSmall peptides have the benefits of small molecule drugs, like aspirin, and large antibody therapies, like rituximab, with fewer drawbsmall molecule drugs, like aspirin, and large antibody therapies, like rituximab, with fewer drawbacks.
With that goal in mind, Xue and his team joined forces with Chen Zhao, MD, PhD, UI assistant professor of pathology, and used their understanding of CML genetics to look for small molecules or drug compounds that might be able to eradicate the leukemia stem ceWith that goal in mind, Xue and his team joined forces with Chen Zhao, MD, PhD, UI assistant professor of pathology, and used their understanding of CML genetics to look for small molecules or drug compounds that might be able to eradicate the leukemia stem cewith Chen Zhao, MD, PhD, UI assistant professor of pathology, and used their understanding of CML genetics to look for small molecules or drug compounds that might be able to eradicate the leukemia stem cells.
The process of drug discovery has been dominated in recent years by the screening of large libraries of structurally diverse small molecules for effects on target proteins associated with disease.
One option would be to shut down the expression of BCL11A, using small pieces of RNA, in blood - forming stem cells that would then be given to a person with the disease; another would be to inhibit the action of BCL11A directly using a more traditional, and possibly more practical, small - molecule drug.
Arguably, the most economical and efficient strategy for development of small molecules with regenerative medicine applications is to use non-mammalian animal models for both target - and phenotype - based drug discovery.
In another experiment, a more direct test of druggability, the researchers used a library of small molecules — broadly representing potential drug molecules — to see which could compete with the probe in binding covalently to reactive lysines on human proteins.
SOUTH PLAINFIELD, NJ — December 6, 2007 — PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of small - molecule drugs targeting post-transcriptional control mechanisms, today announced an expanded research collaboration with the Spinal Muscular Atrophy (SMA) Foundation.
Determining whether a disease - related protein can react strongly with a small - molecule probe or candidate drug — in a way that alters its function — is often an essential first step in drug development.
UniChem supports the integration of high - throughput screening data on small molecules with chemical libraries and chemical resources for drug discovery and optimization.
Using the new method, published on July 31, 2017 in Nature Chemistry, the TSRI scientists identified more than 100 human proteins that are likely to be targetable with small molecule, pill - based 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.
«This partnership puts our mRNA Therapeutics platform in the hands of Karolinska's world - class scientists and clinical researchers to develop new drugs and therapeutic approaches that can not be done with small molecules or biologics — bringing new hope to patients with serious diseases.»
In its inaugural collaboration that focuses on small molecule drug discovery, IACS has signed an exclusive agreement with TESARO, Inc., an oncology - focused biopharmaceutical company.
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.
Joshua is an organic / medicinal chemist with many years of experience in small - molecule drug discovery.
Stanford University School of Medicine Accelerating drug discovery with iPS cells and small molecule screen
Memory improved in mice injected with a small, drug - like molecule discovered by UC San Francisco researchers studying how cells respond to biological stress.
Other companies, including the Swiss drug giants Novartis and Roche, along with Boston's Paratek Pharmaceuticals, are developing ingestible small - molecule drugs designed to increase the inclusion of SMN2 «s exon 7.
Dr. King points out that a number of oral drug candidates inhibit PKC - delta and that it may be possible to suppress PKC - delta production with microRNAs (small RNA molecules that help to regulate protein generation).
«Scientists can now, at the click of a button and at a single online location, rapidly identify the biological and structural characteristics of an actual or potential drug target, find the genes and proteins that it interacts with, assess its structural druggability and identify any small molecule chemical inhibitors.
A number of different strategies are being pursued, including small - molecule drugs and antisense therapies, all with the same goal in mind: to therapeutically boost the levels of full - length SMN protein.
It also raises the possibility to interfere with these systems by any means (small molecules, FDA approved marketed drugs) and evaluate the consequences on both neurogenesis and neuroplasticity.
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 project that is farthest along involves the discovery of small molecules for use in combination with drugs of last resort for extensively drug - resistant tuberculosis infections.
Our goal is to study the molecular mechanisms by which the IL - 1R - TLRs mediate signaling, with the long - term aim of developing more effective anti-inflammatory small - molecule drugs.
As reported online in PLoS One on October 3, Hopkins colorectal cancer specialists John Abraham, Ph.D., and Stephen Meltzer, M.D. - working with the notion that small molecules generally make better treatment packages - designed small bits of protein only 10 amino acids long as the foundation for their drugs.
Performed pro-bono work investigating small cap pharmaceutical companies, ensuring scientific and clinical relevance for target drugs, revenue forecasting, market research for drugs with similar target molecule
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