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Treatment involved editing the patient's DNA using zinc finger nucleases technique. It was carried out on Brian Madeux, a 44 year old man suffering from Hunter syndrome, a metabolic disorder caused by a gene error. The treatment was carried out by Paul Harmatz and his team at UCSF Benioff Children's Hospital. The patient was reported not to have experienced any major side effects after the treatment and no safety issued emerged in subsequent months. 2017-11-16T00:00:00+0000| 16 Nov 2017 | | First patient received therapy involving gene editing inside the bodyHarmatz, Madeux | University of California San Francisco | Gene therapy, Gene editing, Zinc fingers |
Patients with severe haemophilia A received a single infusion of a copy of a missing gene that allows their cells to produce Factor VIII, a protein needed to stop bleeding. This was delivered using an adeno-associated virus vector. The patients were enrolled between September 2015 and April 2016 into one of three dose cohorts at five sites across the UK. The five UK trial sites included: The Royal London, Guys and St Thomas', Birmingham, Cambridge and Hampshire hospitals. At 54 week follow-up 85% of the patients were found to have normal or near normal Factor VIII levels. Thirteen of the patients no longer needed their previously regular treatment. S. Rangarajan, et al, 'AAV5–Factor VIII Gene Transfer in Severe Hemophilia A', New England Journal of Medicine (9 Dec 2017), DOI: 10.1056/NEJMoa1708483.2017-12-09T00:00:00+0000| 9 Dec 2017 | | Gene therapy shown to be safe and efficacious treatment for haemophilia A in British trialsRangarajan, Walsh, Lester, Perry, Madan, Laffan, Hua Yu, Vettermann, Pierce, Wong, Pasi | Barts Health NHS Trust, Queen Mary University, BioMarin Pharmaceutical | Gene therapy |
Developed by Spark Therapeutics and Novartis, the drug (Voretigene neparvovec/Luxturna) aims to correct restore vision in patients born with Leber congenital amaurosis, a rare genetic retinal disease. Those with the condition have mutations in both copies of the RPE65 gene which cause sight loss from an early age and eventually causes blindness. The treatment aims to provide a working copy of the RPE gene.2017-12-19T00:00:00+0000| 19 Dec 2017 | | US FDA approved gene therapy approved to treat rare genetic retinal disease | Novartis, Spark Therapeutics | Gene therapy, Ophthalmology |
2018-01-01T00:00:00+0000| 2018 | | Sharma elected to join the American Society for Clinical InvestigationSharma | | |
C T Charlesworth et al, 'Identification of Pre-Existing Adaptive Immunity to Cas9 Proteins in Humans', bioRXiv (2018), https://doi.org/10.1101/2433452018-01-05T00:00:00+0000| 5 Jan 2018 | | Researchers identify pre-existing antibodies targeting CAS9 proteins raising possibility of immune responses undermining utility of CRISPR-Cas9 for gene therapyCharlesworth, Deshpande, Dever, Dejene,Gomez-Ospina, Mantri, Pavel-Dinu, Camarena, Weinberg, Porteus | Stanford University | Gene therapy, Gene editing, CRISPR-Cas9 |
The test hunts for 16 genes that regularly arise in cancer and 8 proteins released by tumours. It was trialled in 1,005 patients with cancers of the ovary, liver, stomach, pancreas, oesophagus, colon, lung or breast that had not yet spread to other tissues. The work was published in J.D. Cohen et al, Detection and localization of surgically resectable cancers with a multi-analyte blood test', Science, 18 Jan 2018, eaar3247, DOI: 10.1126/science.aar3247
2018-01-17T00:00:00+0000| 17 Jan 2018 | | Blood test detecting mutated DNA and proteins released by tumours shown to pick up early signs of 8 common cancersCohen, Yuxuan Wang, Thoburn, Afsari, Danilova. Douville, Javed, Wong, Mattox, Hruban, Wolfgang, Goggins, Molin, Wang, Roden, Klein, Ptak, Dobbyn, Schaefer, Silliman, Popoli, Vogelstein, Browne, Schoen, Brand, Tie, Gibbs, Wong, Mansfield, Jen, Hanash, Falc | Johns Hopkins University | Diagnostics, Oncology |
Z. Liu et al, 'Cloning of macaque monkeys by somatic cell nuclear transfer', Cell (2018), http://dx.doi.org/10.1016/j.cell.2018.01.020
2018-01-24T00:00:00+0000| 24 Jan 2018 | | Chinese scientists cloned the first monkeys by somatic cell nuclear transferLiu, Cai, Wang, Nie, Zhang, Xu, Xiatong, Zhang, Lu, Poo, Sun | Chinese Academy of Sciences | Cloning |
A team of scientists from the UK, USA and Canada showed the device to be useful in tracing infectious diseases like Ebola and the Zika virus in Africa and the US. The device, called the MiniOn sequencer, was developed by Nick Loman and his doctoral student Josh Quick at the University of Birmingham. The device works by passing long strands of DNA through a tiny hole (the eponymous nanopore). Each of strand transmits a unique electrical signal as it passes through the pore, making it possible sequence the 4 bases of the DNA. The method allows for ultra long sequences of DNA to be worked out, 8,000 times longer than a typical sequencing read. It also makes it possible to sequence previously uncharacterised regions of the genome. Details of the research carried out with device were published in M Jain et a, 'Nanopore sequencing and assembly of a human genome with ultra-long reads', Nature Biotechnology, 36 (2018), 338-45.2018-01-29T00:00:00+0000| 29 Jan 2018 | | Handheld device using nanotechnology shown to be promising tool for sequencing human genomeLoman, Quick, Jain, Koren, Miga, Rand, Sasani, Tyson, Beggs, Dilthey, Fiddes, Malla, Marriot, Nieto, O'Grady, Olsen, Pedersen, Rhie, Richardson, Quinlan, Snutch, Tee, Paten, Philippy, Simpson, Loose | University of Birmingham, University of Nottingham, University of Utah, University of British Columbia, University of East Anglia, Ontario Institute for Cancer Research, University of California Santa Cruz, National Human Genome Research Institute | DNA sequencing, Genomics |
The work was undertaken by a group of researchers at the University of Edinburgh led by Evelyn Telfer. It involved taking tiny pieces of ovarian tissue from 10 women undergoing elective caesarean surgery extracting priorial follicles, small structures that have the potential to release an egg, which were then placed in a nutrient-rich liquid to grow. The team then carefully removed the fragile, immature eggs and some surrounding cells from the follicles and placed them on a special membrane with the addition of growth-supporting proteins so that they could grow to become the size you would see of an egg during ovulation. Most of the eggs failed to grow, but 10% completed their journey to maturity - that is they were able to divide and halve their chromosomes so they were ready to be fertilised by sperm. The work was published in M McLaughlin, DF Albertini, WHB Wallace, RA Anderson, EE Telfer, Molecular Human Reproduction, 24/3 (March 2018) 135-42. DOI: 10.1093/molehr/gay002. 2018-01-30T00:00:00+0000| 30 Jan 2018 | | First human eggs grown in laboratoryTelfer, McLaughlin, Albertini, Wallace, Anderson | University of Edinburg | Reproduction |
2018-02-01T00:00:00+0000| Feb 2018 | | Hepatitis B Foundation Drug Watch listed 36 drugs at various development stages for chronic hepatitis B | | |
The drug was developed by Seattle Genetics with Astellas Pharma. 2018-03-01T00:00:00+0000| Mar 2018 | | FDA granted drug breakthrough therapy designation to enfortumab vedotin for treating metastatic urothelial cancer | Seattle Genetics, Astellas Pharma | Monoclonal antibodies, Oncology |
First discovered by Array Biopharma and then licensed to Cascadian Therapeutics, tucatinib is designed to target HER2 (human epidermal growth factor receptor 2) ‘tyrosine kinase inhibitor’ (TKI), an enzyme that helps to promote the growth of tumors in HER2 positive cancers.2018-03-01T00:00:00+0000| Mar 2018 | | Seattle paid $614.1 million to acquire rights to tucatinib from Cascadian Therapeutics | Seattle Genetics, Cascadian Therapeutics | Oncology |
A randomised pilot study was run to assess two possible routes of FMT delivery, colonic and nasogastric, in patients with ulcerative colitis. The trial sought to determine the optimum route of FMT delivery for the treatment of UC and the efficacy of such treatment.2018-03-01T00:00:00+0000| Mar 2018 - Apr 2019 | | STOP-Colitis Pilot Trial | University of Birmingham | Faecal microbiota transplant |
Sulston was a biologist. He played a central role in sequencing the genome of the Caenorhabditis elegans, a transparent nematode (roundworm). It was the first animal to have its genome sequenced. Based on his work with the nematode Sulston helped set up the project to sequence the human genome which he did as director of the Sanger Centre. The first draft of the human genome sequence was completed in 2000. Sulston shared the Nobel Prize in 2002 for identifying how genes regulate the life cycle of cells through apoptosis. 2018-03-09T00:00:00+0000| 9 Mar 2018 | | John E Sulson diedSulston | Laboratory of Molecular Biology, Sanger Institute | Cell, Genetics, DNA sequencing |
Douglas Waters, an 86 year old patient at Moorfield Eye Hospital in London gained improved eyesight in his right eye after having stem cells inserted underneath the rods and cones in the back of his eye. The stem cells were grown in a patch and were designed to provide new retinal pigment epithelium. While the patient's eyesight was not completely restored, he gained a renewed ability to read the newspaper. Another patient with the same condition who received the same treatment also experienced some improvements to her eyesight. Both patients, who were taking part in a phase I trial, maintained improved vision for a year after the treatment. The results were published in L da Cruz, K Fynes, O Georgiadis et al, 'Phase 1 clinical study of an embryonic stem cell–derived retinal pigment epithelium patch in age-related macular degeneration', Nature Biotechnology, 36 (2018) 328-37. 2018-03-19T00:00:00+0000| 19 Mar 2018 | | Stem cell treatment reported to be promising treatment for age-related macular degenerationda Cruz, Fynes, Georgiadis, Kerby, Luo, Ahmado, Vernon, Coffey | University College London | Stem cells |
The aim of the drug is to mute hepatitis B genes to give the immune system a chance to fight the hepatitis B infection.2018-03-27T00:00:00+0000| 27 Mar 2018 | | Dosing trial launched with humans to test use of RNA interference drug to treat hepatitis B | Arrowhead Pharmaceuticals | RNA interference |
Twenty-two patients were treated in the trial over 42 months in six centres around the world. The treatment consisted of taking immature stem cells from the patient's bone marrow and using a harmless virus to infect the cells with a copy of the normal globin gene. The new genetically altered cells were then reintroduced into the patient's bloodstream after their marrow had been cleared of diseased cells using chemotherapy. Nine out of the twenty-two patients severe beta thalassemia were able to cut down on the number of transfusions they needed by 74%, with three of them no longer needing any transfusions at all. The same was true of the twelve out of thirteen patients with less severe thalassemia. A A Thompson et al, 'Gene therapy in patients with transfusion-dependent beta thalassemia', New England Journal of Medicine, 378 (2018), 1479-93.2018-04-19T00:00:00+0000| 19 Apr 2018 | | Gene therapy shown to be promising treatment in clinical trials for beta thalassemiaThompson, Walters, Kwiatkowski, Rasko, Ribeil, Hongeng, Magrin, Schiller, Payen, Smeraro, Moshous, Lefrer | North Western University, University of California San Francisco, University of California Los Angeles, University of Sydney, University of Paris, Harvard University, Mahidol University, German Cancer Research Centre | Gene therapy |
Developed by Arrowhead Pharmaceuticals, the drug, ARC-520, is administered with the antiviral entecavir. The treatment cleared the HBsAg from one patient and substantially reduced it in the remaining patients. The results were presented to the European Association for the Study of the Liver. 2018-05-02T00:00:00+0000| 2 May 2018 | | Positive results for RNAi drug for treating chronic hepatitis b reported from 8-person clinical trial | Arrowhead Pharmaceuticals | RNA interference |
Falkow was an American microbiologist who made his scientific mark by showing how bacteria develop resistance to antibiotics. During the 1960s he demonstrated that bacteria could acquire resistance by swapping genetic material via plasmids, small microbial DNA molecules. Thereafter he focused his attention on how pathogens cause disease and in 1985 helped to identify a single genetic locus in Yersinia pseudotuberculosis, a Gram-negative bacteria, that accounts for its ability to infect cultured animal cells. He later showed that a sub-type of E. coli caused a life-threatening diarrhoea prevalent in many low-income countries. Known as the founder of molecular pathogenesis, Falkow's work paved the way to the development of new vaccines, including for whooping cough. He also helped to devise a uniform nomenclature for bacterial plasmids2018-05-05T00:00:00+0000| 5 May 2018 | | Stanley Falkow diedFalkow | Georgetown University School of Medicine, University of Washington School of Medicine, Stanford University | Antimicrobial resistance, Plasmids |
Skou was a Danish biochemist who was awarded the Nobel Prize for Chemistry in 1997 for discovering an ion-transporting enzyme called sodium-potassium-activated adenosine triphosphatase (Na+-K+ ATPase), which is found in the plasma membrane of animal cells. The enzyme is important to the transport of molecules through a cell's membrane. Skou made the discovery in the late 1950s. A number of other similar ATPase-based enzymes were discovered later, including one that helps control muscle contraction.2018-05-28T00:00:00+0000| 28 May 2018 | | Jens C Skou diedSkou | Aarhus University | Cell, Biochemistry |
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