New Viral Vector for Sickle Cell Gene Therapy Likely to Be More Effective, NIH Study Says – Sickle Cell Anemia News

A newly designed viral vector the vehicle that delivers a gene therapyto a patients cells for use insickle cell anemia is more efficient than earlier vectors at introducing healthy copies of genes into stem cells and can be produced in greater amounts, studies in animal models show.

The study Development of a forward-orientated therapeutic lentiviral vector for hemoglobin disorders was published in the journal Nature Communications.

Hemoglobin is the protein in red blood cells that binds oxygen, allowing oxygen to be transported around the body. Mutations in the HBBgene, which encodes a component of hemoglobin, causessickle cell.

Gene therapies involve either altering the mutated gene or introducing a healthy version of that gene to the body. Still under development for sickle cell, an estimated 27 patients have undergone experimental gene therapy. One strategy involves removing hematopoietic stem cells (which function to produce blood cells) from a patients bone marrow. A healthy copy of the HBB gene is then introduced into the cells using a modified, harmless virus known as a viral vector. The cells are then transplanted back into the patient where they will produce healthy red blood cells.

Traditionally, viral vectors for sickle cell have been designed in a way known as reverse structural orientation. This means that the HBB gene is translated or read from right to left, like reading an English sentence backwards. The reverse structural orientation design ensures that a key section of the gene (known as intron 2), which is necessary for the production of high levels of the HBB gene, is retained during viral vector preparation.

However, this design makes preparing the viral vectors more difficult, and decreases the efficiency of introducing the gene into the stem cells.

Researchersat the National Institutes of Healthdesigned a new viral vector, one in which the HBB gene is forward orientated and read from left to right. Genes essential for the virus were inserted into intron 2, meaning that only vectors that retained intron 2 would be produced (a type of positive selection).

Our new vector is an important breakthrough in the field of gene therapy for sickle cell disease, John Tisdale, MD, chief of the Cellular and Molecular Therapeutic Branch at the National Heart, Lung, and Blood Institute (NHLBI) and the studys senior author, said in a press release.

Its the new kid on the block and represents a substantial improvement in our ability to produce high capacity, high efficiency vectors for treating this devastating disorder, he added.

The researchers compared the new vectors to traditional reverse-orientated vectors in mouse and monkey models. The new vectors were four to 10 times more efficient at introducing the healthy HBBgene into the stem cells, and could carry up to six times more HBB genes compared to the conventional vectors.

Furthermore, the new vectors remained incorporated into the cells of monkeys up to four years after a transplant. These vectors could also be produced in greater amounts, which may lessen the time and costs required for large-scale vector production.

The researchers hope that these characteristics will make gene therapy for sickle cell disease more effective and increase its use. The new vector design still needs to be tested in clinical trials in patients.

Our lab has been working on improving beta-globin vectors for almost a decade and finally decided to try something radically different and it worked, Tisdale said.

These findings bring us closer to a curative gene therapy approach for hemoglobin disorders, he added.

Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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New Viral Vector for Sickle Cell Gene Therapy Likely to Be More Effective, NIH Study Says - Sickle Cell Anemia News

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Caroline Wyatt: The fight to reverse damage caused by MS – BBC News

Image caption Caroline Wyatt visited Prof Robin Franklin to find out more about a drug that might help stop the progression of MS

"I don't like to think of the future. It's such a big question mark. I just keep living in the present."

Karine Mather was diagnosed with MS when she was 27, although she noticed the first symptoms much earlier.

It started off as a mental-health issue with anxiety and depression, she remembers. Later, she noticed she was starting to limp when she walked longer distances.

Karine began using a walker to help with her balance and stamina, and then a scooter when she could no longer walk very far.

"I got to the stage where the wheelchair became quite liberating, and gave me back a sense of freedom again. Now I rely on the power-chair full-time because I can't stand by myself any more."

Now Karine and her wife, Sarah, have had to give up their full-time jobs.

Karine was forced to stop working as a customer service adviser at a bank because she could no longer fulfil the physical demands of work and Sarah gave up working as a data analyst so she could take care of Karine.

Now 34, Karine retains the use of just one hand, and suffers pain, stiffness and spasticity in her body that has got worse as the disease has progressed.

"It feels like a fist clenching all the time. And I have days when my mind is cloudy and I miss out words and sentences."

Both remain upbeat but the financial, as well as the emotional, impact of MS has been huge.

Karine's MS is the type known as "primary progressive", or PPMS, which meant that for the first years after diagnosis, no disease-modifying treatment was available.

One new drug - Ocrevus, or ocrelizumab - was recently licensed for early PPMS in the UK but came too late to help Karine.

Now the MS Society is launching an ambitious "Stop MS" appeal, aiming to raise 100m to fund research over the next decade into treatments that can stop the progression of disability in MS.

Since being diagnosed with MS in 2015, after many years of symptoms, I've been looking for anything that might help slow or even stop the progression of my MS, which affects the nerves in my brain and spinal cord.

I last wrote about my MS after travelling to Mexico for an autologous stem cell transplant (aHSCT) in 2017.

Sadly, despite initial improvements, I'm now back to where I was before: slowly but surely getting worse.

The only improvements that have endured are the lifting of some of the crushing brain fog I had before HSCT and less hesitation in my speech.

For both, I am eternally grateful, as they mean I can continue to work at the BBC, in the job I love.

However, I have no idea how long this reprieve will last.

The fatigue that had long been my worst symptom is now back with a vengeance, so that staying awake throughout a busy working day remains a challenge.

That MS fatigue did lift for a few months, and it felt miraculous. I awoke every day refreshed. But then it returned, and I awake after eight full hours fast asleep feeling as if I haven't been to bed at all.

The ageing process - including menopause - has almost certainly been a factor in the worsening of some symptoms.

Ageing cells repair less well, and with my faulty immune system apparently determined to keep stripping away the myelin sheath that should protect my nerves, I'm less able now to repair the damage than I was when the disease first began to affect me in around 1992.

Since 2016, I've had to walk using a stick to aid my balance. It is sparkly-topped; an effort to make the accoutrements of disability just a little more cheery.

Dizziness is now a constant companion. It rarely goes away, making car travel or even buses a nightmare. Just turning my head too fast can make me stagger or fall over.

And for the past year or two, my right foot has begun to drag along the ground thanks to foot drop, meaning that I trip more often because I can't fully raise it.

I am always grateful to the strangers who kindly stop to help me up from the uneven pavement when I do fall.

Perhaps most worrying for me is that my right hand no longer works as it used to, catching on the computer keyboard as my outer fingers drag lazily along the keys, sullenly refusing my brain's command to lift.

In the mornings, both my hands and my feet are numb and frozen, then painfully full of pins and needles before warming up enough to be usable a few hours later.

When I wake, I wonder how long it might be until these hands and feet barely function at all, and quickly push that unwelcome thought away.

I'm well aware how very lucky I am that the progression of my MS has been relatively slow - at least until recently. I've learned how better to conserve energy for the things that really matter, though I still chafe at how little I manage to achieve.

Having enough energy to cook a meal from scratch on a day off is a cause for rejoicing. I'm still learning how to save up enough energy for family and friends, and not use up all of my much-depleted ration for work or research.

I have had to face the fact that I have now probably gone from the relapsing-remitting phase of MS (for which a dozen or so treatments exist) into the secondary progressive phase, for which there is currently no treatment licensed in the UK to stop the relentless progression that will affect so many of the 100,000 or more of us living with MS here.

But that may be about to change.

Anna Williams, professor of regenerative neurology at the University of Edinburgh, is looking at how the brain responds to MS damage and how the fatty myelin sheath under attack in MS can be restored more efficiently.

"We have to look at ways to stop the nerves dying," she says. "We want to be able to try to limit that either by keeping the nerves alive, or keeping them working better."

Repurposing existing drugs to help with remyelination should prove the quickest route to therapies for progressive forms of MS, because creating and licensing new ones is a much lengthier and more expensive process.

Prof Williams still sees patients at the Anne Rowling Clinic of Regenerative Neurology in Edinburgh, named in memory of the Harry Potter author J K Rowling's mother, who had MS. (The author this year donated 15m for research at the unit.)

"At the moment, with PPMS or SPMS, we can always give relief for pain or stiffness but we won't change the course of the disease.

"So for those patients, to slow or stop or reverse the disease can only be done with more research, and money is critical for research."

The biggest trial yet in the UK for patients with secondary progressive MS is the MS STAT2 trial, conducted by Prof Jeremy Chataway for the UCL Queen Square Institute of Neurology in London.

The trial is still recruiting at 30 centres across the UK to look at whether simvastatin, a drug used to treat high cholesterol, can slow or stop disability progression. If so, it has the potential to become one of the first disease-modifying therapies for people with secondary progressive MS.

And perhaps most encouraging of all, Prof Robin Franklin and his team at the Wellcome-MRC Cambridge Stem Cell Institute recently published research suggesting a common diabetes drug - metformin - could hold the key to stopping disease progression in MS.

Costing just a few pence per tablet, metformin appears to have an ability to restore cells to a younger, healthier state and encourage myelin regrowth.

The next question is whether it works in people as well as it does in the lab.

Prof Franklin says: "This is a drug that's well tolerated and widely available. There is every reason to believe that the effects that we have seen - which have been so spectacular - will translate into humans.

"This is the great frontier of MS therapy. We're good at stopping the inflammation in MS. What we're not so good at doing is repairing the damage. All this work has given us some real hope that this medicine will reverse the damage done by MS."

I certainly feel rather more hopeful than I did.

I've changed as much about my lifestyle as I can - prioritising sleep, eating healthily, largely giving up alcohol, doing yoga and stretching every day, and cutting back on stress, be that reporting from war zones or attending too many BBC meetings.

But I'm all too aware that time is against me as my ageing brain and body struggle to repair the damage done in their lengthy continuing battle with my own immune system.

My hope now is that these trials will show good enough results in the next few years for at least one or two of the drugs to be rapidly approved for MS so they can help people like Karine and me before it's too late.

I ask Karine what she makes of the current research.

She is suitably succinct.

"I'm sitting here with just the one limb working and I'm thinking - quicker, please."

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Caroline Wyatt: The fight to reverse damage caused by MS - BBC News

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Global 3D Bioprinting Market Outlook, 2019-2024 – Market Anticipated to Reach $1.64 Billion by 2024 – – Business Wire

DUBLIN--(BUSINESS WIRE)--The "3D Bioprinting Market by Component (3D Bioprinters (Microextrusion, Inkjet, Laser), Bioink (Natural, Synthetic, Hybrid)), Material (Hydrogel, Living Cells), Application (Skin, Drug Research), End user (Biopharma, Academia) - Global Forecast to 2024" report has been added to's offering.

The 3D bioprinting market is projected to reach USD 1,647 million by 2024 from USD 651 million in 2019, at a CAGR of 20.4% from 2019 to 2024.

The growth in this market is mainly driven by technological advancements in 3D bioprinters and biomaterials, increasing the use of 3D bioprinting in the pharmaceutical and cosmetology industries, and rising public and private funding to support bioprinting research activities. On the other hand, a shortage of skilled professionals and high development and production costs are hampering the growth of this market.

The major players in the market include Organovo Holdings Inc. (US), CELLINK (Sweden), Allevi Inc. (US), Aspect Biosystems Ltd. (Canada), EnvisionTEC GmbH (Germany), Cyfuse Biomedical K.K. (Japan), Poietis (France), TeVido BioDevices (US), Nano3D Biosciences, Inc. (US), ROKIT Healthcare (South Korea), Digilab Inc. (US), regenHU (Switzerland), GeSiM (Germany), Advanced Solutions Life Sciences (US), and Regenovo Biotechnology Co., Ltd. (China) among others.

Microextrusion technology commanded the largest share of 3D bioprinters segment owing to technological advancements

The component segment of the 3D bioprinting market is segmented into 3D bioprinters and bioinks. The 3D bioprinters market is further sub-segmented on the basis of technology into magnetic 3D bioprinting, laser-assisted bioprinting, inkjet 3D bioprinting, microextrusion bioprinting, and other technologies; whereas bioinks segment is further sub-segmented into natural, synthetic, and hybrid bioinks. The microextrusion bioprinting technology has commanded the largest share of the market in 2019 due to technological advancements in the segment and the increasing research activities.

The drug discovery research application segment accounted for the largest share of the 3D bioprinting market in 2019

In terms of applications, the 3D bioprinting market is segmented into research applications and clinical applications. The demand for research applications is further sub-segmented into drug research, regenerative medicine, and 3D cell culture. Among these, the drug research segment accounted for the largest share of the market in 2019, owing to the growing adoption of 3D bioprinting technology by biopharmaceutical companies. While, in terms of clinical applications, the market is segmented into skin, bone & cartilage, blood vessels, and other clinical applications.

Based on material, living cells segment commanded the leading market share in 2019

Based on material, the 3D bioprinting market is broadly segmented into hydrogels, extracellular matrices, living cells, and other biomaterials. Increasing R&D activities for the use of living cells in 3D bioprinting is driving the growth of the living cells segment. Living cells have the ability to fabricate patient-specific tissues in a defined manner. With advances in 3D bioprinting, scientists and researchers are making use of living cells as a biomaterial in 3D bioprinting. These cells can be used to print living tissues as well as organ structures for surgical implantations. However, ethical issues associated with the use of stem cells in 3D bioprinting might hamper the growth of the segment.

The US 3D bioprinting market to hold prominent market share over the forecast period

On the basis of region, the 3D bioprinting market is segmented into North America, Europe, Asia Pacific, and Rest of the World (Latin America, and the Middle East and Africa). The US held a significant share of the global 3D bioprinting market in 2019. Factors such as new product launches and technological advancements in 3D bioprinting technology and the presence of key players in the region are driving the growth of the 3D bioprinting market in the US. Moreover, extensive research activities and funding for 3D bioprinting will further fuel the market growth in the US.

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Global 3D Bioprinting Market Outlook, 2019-2024 - Market Anticipated to Reach $1.64 Billion by 2024 - - Business Wire

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Cell Culture Protein Surface Coatings Market will Going to be Worth US$ 623.4 Mn by 2020 – Online News Guru

Cell culture is a complex procedure in which cells are grown under controlled physical conditions outside the natural environment. These cells are used to develop model systems for research, study of cellular structure and functions, stem cell research, drug discovery and genetic engineering. Growing scope of cell culture and its applications has led to increased use of protein coated surfaces, as these provide better adhesion and proper nutrition for growth of the cells during cell culture.

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Rising investment by government and market players in stem cell research and development activities is one of the major factors driving the cell culture protein surface coatings market. Becton, Dickinson and Company grants a total of USD 100,000 worth reagents every year to 10 scientists pursuing research activities in stem cells. Similarly, the European Union funded four stem cell research projects in its Seventh Framework Program for Research and Technological Development (2007 2013).

High funding is leading to extensive stem cell research, resulting in increased use of cell culture protein surface coating products. Moreover, diverse applications of stem cells such as development of bone grafts and artificial tissue would fuel the demand for cell culture protein surface coatings during the forecast period. In addition, increasing cell culture applications in toxicology studies and cell-based assays would boost the demand for protein surface coating products. Currently, 2D cell culture is the most preferred technique by researchers worldwide due to lack of compelling data to switch to 3D cell culture.

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Based on the types of coating, the self-coating segment held the majority share of the total market in 2013 in comparison to pre-coating segment. The pre-coating segment is sub-segmented on the basis of different labware such as slides, micro-well/multi-well plates, flasks, petri dishes and cover slips. In terms of revenue, the micro-well/multi-well plates segment held the largest share of the total pre-coating market in 2013. The cell culture protein surface coatings market is also differentiated on the basis of protein sources, which include plant, animal, human and synthetic. The synthetic protein source segment is expected to grow at a faster rate in the global cell culture protein surface coatings market during the forecast period. Growth of the synthetic protein source segment is attributed to the rising demand for animal-free coating surfaces in North America and Europe and better attachment profile of poly-L-lysine and poly-L-ornithine.

Geographically, North America and Europe dominated the cell culture protein surface coatings market in 2013 due to large-scale stem cell research activities and rapid adoption of advanced tools for cell culture. However, Asia Pacific is expected to grow at the highest CAGR due to the presence of untapped opportunities, increasing drug discovery activities, impressive development of healthcare and biotechnology infrastructure and growing trend among the market players to expand business in the region.

The global cell culture protein surface coatings market is characterized by the presence of few big key players such as Corning Incorporated, Greiner Bio-One International AG, Merck Millipore, Sigma-Aldrich Corporation and Thermo Fisher Scientific, Inc. Cornings acquisition of Becton Dickinson & Company has helped it to gain a dominant share in the global cell culture protein surface coatings market. Competition among these market players is high, which induces them to develop new and better protein surface coatings for cell culture and associated applications.

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Cell Culture Protein Surface Coatings Market will Going to be Worth US$ 623.4 Mn by 2020 - Online News Guru

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Six people with links to UCLH listed among most influential people in London – University College London Hospitals

The Progress 1000 is compiled every year. The theme of this years list is the future and technology, and embraces whole new sectors including augmented and virtual reality and cyber security, as well as a wide range of activists challenging inequality and helping the environment.

Dr Prasanna Sooriakumaran, a consultant prostate cancer surgeon at UCLH, was included for his pioneering robotic surgery. He is investigating new techniques to spare men with prostate cancer the potentially debilitating effects of surgery. Early trials show that his new technique has drastically reduced recovery time for up to 94 per cent of patients.

Prasanna said: It is a great honour to top the list of Londons most influential doctors in the Progress 1000 after having also made the list last year. This is a testament to the wonderful staff at UCLH, who provide world class care to men with prostate cancer.

Professor Bryan Williams is director of one of the UKs leading NIHR Biomedical Research Centres at UCLH, director of research at UCLH and Chair of Medicine at UCL. He is a clinician at UCLH and is recognised as one of the worlds leading authorities on high blood pressure.

He said: It is good to see recognition of the influence that staff at UCLH have in driving forward medical research and innovation in London and beyond.

Professor Charles Swanton, UCLs professor of personalised cancer medicine with a lab at UCL Cancer Institute and the Francis Crick Institute in Kings Cross, a consultant at UCLH and chief clinician at Cancer Research UK, is leading pioneering research on lung cancer. Professor Swanton studies how cancers evolve in the body to spread and become resistant to therapy. He is also researching ways to treat tumours more effectively.

Charlie said: This is a great testament to the hospital, university, Crick and CRUK and the team for making TRACERx possible.

Professor Tariq Enver, director at UCL Cancer Institute and professor of stem cell biology at UCL, leads a grand coalition in the war on cancer by encouraging closer working relations between UCL, Kings College London, Queen Mary University of London and the Francis Crick Institute, creating a centre of excellence for biotherapeutics.

Tariq said: It is fantastic that the vital work being done by the team in London - which has the potential to transform cancer treatment in the long run is being recognised and encouraged. Being nominated is a massive boost for all of us who have worked so hard to reach this point.

Professor Ravi Gupta, until recently an infectious diseases clinician at UCLHs Hospital for Tropical Diseases, studies the evolution and spread of HIV drug resistance globally. He, along with colleagues at Imperial College London, recently published a report on how a patient with HIV and lymphoma is now free from both conditions after an allogeneic stem cell transplant using cells from a donor lacking a critical receptor protein for HIV infection, CCR5. This work has rejuvenated the field of HIV gene therapy.

He said: It has been humbling to work at UCLH alongside such dedicated staff, both clinical and academic. I hope that recognition of the London Patient HIV cure in the Progress 1000 list will serve as an inspiration in London and beyond in the fight against HIV/AIDS.

Professor Chris Whitty, the chief medical officer for England and a UCLH physician, was included on the list for his work which focuses on the diagnosis and management of infectious diseases in children and adults.

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Six people with links to UCLH listed among most influential people in London - University College London Hospitals

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