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bioquick news--life science news from around the globe news about us bioquick japanese edition a-t children’s project announces selection of first child to receive aso gene therapy for ataxia-telangiectasia sun, 12/09/2018 - 09:51 — bioquicknews on november 30, 2018, the ataxia-telangiectasia (a-t) children’s project and its president brad margus announced that the project has selected a little, 18-month girl on the west coast of the u.s. to be the first a-t child in history to receive gene therapy. the project had the pleasure of letting th girl's parents know the exciting news last week. how was the first child selected? the laboratory of dr. tim yu at boston children’s hospital/harvard collected and grew skin, blood, and stem cells from three different young children who we believed had the right type of mutations in their a-t genes to be treated with an antisense oligonucleotide (aso) gene therapy approach. the scientists then made many different oligonucleotides designed to silence each of the children’s mutations and tested them in each child’s cells. in the end, the group identified several oligonucleotide molecules that worked really well in this little girl’s cells, not only silencing her mutation and causing the a-t protein to be made correctly, but also causing downstream biological pathways to be activated as though her cells were from a healthy child (confirming this downstream function took us longer than planned to do, but now gives us more confidence to move forward). this means that we now have both our drug and our patient selected. very soon, we’ll be signing a contract to begin the manufacture of enough quantity of the drug to run our “n of 1” clinical trial (we’ll keep the other oligonucleotides that worked as backups). we’ll also make sure that the drug produced will be clinical-grade to satisfy the fda. as soon as we receive the first batch of this final version of the drug, we’ll test it for safety in rats before applying to the fda for approval to start testing it in a human. read more sugars & microbiome in mother's milk influence neonatal rotavirus infection tue, 11/27/2018 - 19:21 — bioquicknews using a multidisciplinary approach, an international team of researchers from several institutions, including baylor college of medicine, reveals that complex interactions between sugars and the microbiome in human milk influence neonatal rotavirus infection. reported online on november 27, 2018 in the journal nature communications, this study provides new understanding of rotavirus infections in newborns and identifies maternal components that could improve the performance of live, attenuated rotavirus vaccines. the open-access article is titled “human milk oligosaccharides, milk microbiome and infant gut microbiome modulate neonatal rotavirus infection.” "rotavirus infection causes diarrhea and vomiting primarily in children younger than 5, with the exception of babies younger than 28 days of age, who usually have no symptoms. however, in some places, infections in newborns are associated with severe gastrointestinal problems. what factors mediate differences between newborns with and without symptoms are not clearly understood," said first and corresponding author dr. sasirekha ramani (photo), assistant professor of molecular virology and microbiology at baylor college of medicine. "we began our investigation years ago by determining that a particular strain of rotavirus was associated with both asymptomatic infections and clinical symptoms in newborns." dr. ramani and her colleagues first looked for answers from the perspective of the virus. they investigated whether factors such as the amount of virus in newborns or the genome of the virus could be linked to the presence of symptoms in newborns, but did not find any connection between those factors. the researchers then posed the question from the perspective of the newborn. read more international society for extracellular vesicles (isev) releases 2018 update of 2014 guidelines for studies of extracellular vesicles tue, 11/27/2018 - 11:24 — bioquicknews on november 23, 2018, the international society for extracellular vesicles (isev) published online an open-access position paper titled “minimal information for studies of extracellular vesicles 2018 (misev2018); a position statement of the international society for extracellular vesicles and update of the misev2014 guidelines.” the article is scheduled for hard-copy publication in volume 8, issue 1, of the 2019 journal of extracellular vesicles. in the article abstract, the authors note the following: “the last decade has seen a sharp increase in the number of scientific publications describing physiological and pathological functions of extracellular vesicles (evs), a collective term covering various subtypes of cell-released, membranous structures, called exosomes, microvesicles, microparticles, ectosomes, oncosomes, apoptotic bodies, and many other names. however, specific issues arise when working with these entities, whose size and amount often make them difficult to obtain as relatively pure preparations, and to characterize properly. the isev proposed minimal information for studies of extracellular vesicles (“misev”) guidelines for the field in 2014. we now update these “misev2014” guidelines based on evolution of the collective knowledge in the last four years. an important point to consider is that ascribing a specific function to evs in general, or to subtypes of evs, requires reporting of specific information beyond mere description of function in a crude, potentially contaminated, and heterogeneous preparation. read more researching rare genetic disease, scientists uncover key immune regulator tue, 11/27/2018 - 08:51 — bioquicknews scientists at scripps research in california have found an important immune system-regulating protein that in principle could be targeted to treat cancers and chronic viral infections. in a study published online on november 12, 2018 in nature chemical biology, the scientists set out to determine the function of a protein, abhd12 (abhydrolase domain containing protein 12), whose absence causes a rare genetic disease featuring a host of brain and nerve problems. the article is titled “selective blockade of the lyso-ps lipase abhd12 stimulates immune responses in vivo.” the researchers found that abhd12 normally acts as a powerful "brake" on the immune system to keep it from becoming harmfully overactive. mice engineered without the protein have signs of elevated inflammation, and their immune systems are more likely to overreact to a viral infection. the discovery suggests that the absence of abhd12 in people with mutant versions of its gene may cause neurological disease at least in part via excessive immune activity. it also indicates that abhd12 may be a useful target for drugs that boost the immune system--for example against cancers and viruses that normally persist by shutting down people's immune defenses. "this is a good example of how the study of a rare genetic disease can reveal a pathway that plays a key role in human biology," says study co-senior author benjamin cravatt, phd, professor and chair of the department of chemical physiology at scripps research. the rare disease in this case is a mix of progressive brain, peripheral nerve, and eye problems that scientists have given the acronym pharc (polyneuropathy, hearing loss, ataxia, retinitis pigmentosa, and cataract). since 2010, researchers have known that pharc is caused by gene mutations that prevent abhd12 from being made. read more fda approves actpen for genentech’s actemra, a single-dose, prefilled autoinjector for treatment of rheumatoid arthritis, giant cell arteritis, & two forms of juvenile arthritis mon, 11/26/2018 - 21:14 — bioquicknews on november 26,2018, genentech, a member of the roche group (six: ro, rog; otcqx: rhhby), announced that the u.s. food and drug administration (fda) has approved act

URL analysis for bioquicknews.com

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Domain Name: BIOQUICKNEWS.COM
Registry Domain ID: 219279375_DOMAIN_COM-VRSN
Registrar WHOIS Server: whois.register.com
Registrar URL: http://www.register.com
Updated Date: 2016-10-03T22:02:55Z
Creation Date: 2005-09-30T18:02:57Z
Registry Expiry Date: 2018-09-30T18:02:57Z
Registrar: Register.com, Inc.
Registrar IANA ID: 9
Registrar Abuse Contact Email: [email protected]
Registrar Abuse Contact Phone: +1.8003337680
Domain Status: clientTransferProhibited https://icann.org/epp#clientTransferProhibited
Name Server: DNS010.D.REGISTER.COM
Name Server: DNS032.C.REGISTER.COM
Name Server: DNS122.B.REGISTER.COM
Name Server: DNS126.A.REGISTER.COM
DNSSEC: unsigned
URL of the ICANN Whois Inaccuracy Complaint Form: https://www.icann.org/wicf/
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  REGISTRAR Register.com, Inc.

SERVERS

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