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In utero transfusion gives meaningful survival to fetuses with alpha thalassemia major
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This resource is a video abstract of a research paper created by Research Square on behalf of its authors. It provides a synopsis that's easy to understand, and can be used to introduce the topics it covers to students, researchers, and the general public. The video's transcript is also provided in full, with a portion provided below for preview:

"No pregnant woman wants to hear that her developing baby has a life-threatening genetic disease. Historically, women carrying babies with alpha thalassemia major, a type of hereditary anemia, faced the difficult choice between terminating a pregnancy or continuing on despite nearly assured fetal death. Now, researchers at UCSF have reported another option: in utero blood transfusion, or IUT. In this procedure, healthy red blood cells are infused into the fetus, which can reverse the effects of ATM and increase the chance of survival. Fetal hemoglobin – a protein with two alpha and two gamma subunits – is the main oxygen supplier in utero. Patients with ATM lack alpha subunits. As a result, their hemoglobin holds oxygen so tightly that it cannot be released into developing tissues. While a lack of oxygen is harmful at any age, the effects in utero are particularly severe – depriving a developing brain of oxygen, for example, can cause devastating neurologic injury..."

The rest of the transcript, along with a link to the research itself, is available on the resource itself.

Subject:
Applied Science
Health, Medicine and Nursing
Material Type:
Diagram/Illustration
Reading
Provider Set:
Video Bytes
Date Added:
09/20/2019
Oral eliglustat maintains efficacy over 8 years in previously untreated adults with moderate to severe Gaucher disease type 1
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This resource is a video abstract of a research paper created by Research Square on behalf of its authors. It provides a synopsis that's easy to understand, and can be used to introduce the topics it covers to students, researchers, and the general public. The video's transcript is also provided in full, with a portion provided below for preview:

"A recently completed clinical trial of the oral drug eliglustat has delivered promising long-term results for adults with Gaucher disease type 1 – a rare and sometimes life-threatening genetic disorder that interferes with the breakdown of certain types of lipids. GD1 is caused by deficient activity of the lysosomal enzyme acid β-glucosidase. Reduced catalytic activity of the enzyme results in pathogenic accumulation of the enzyme’s substrates, primarily glucosylceramide, in various organs. The result is progressive and debilitating enlargement of the spleen and liver, anemia, low platelet counts, and skeletal manifestations. The historical standard of care is biweekly intravenous infusions of recombinant enzyme, which boosts degradation of glucosylceramide. By contrast, eliglustat, an oral substrate reduction therapy, reduces glucosylceramide storage by slowing its production..."

The rest of the transcript, along with a link to the research itself, is available on the resource itself.

Subject:
Applied Science
Health, Medicine and Nursing
Material Type:
Diagram/Illustration
Reading
Provider:
Research Square
Provider Set:
Video Bytes
Date Added:
02/14/2020
Safety and tolerability of prexasertib in Japanese patients with advanced solid tumors
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CC BY
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This resource is a video abstract of a research paper created by Research Square on behalf of its authors. It provides a synopsis that's easy to understand, and can be used to introduce the topics it covers to students, researchers, and the general public. The video's transcript is also provided in full, with a portion provided below for preview:

"A recent report in the journal Cancer Science adds to the growing body of evidence demonstrating that prexasertib is a promising option for several types of cancer. Two prior clinical trials conducted in the United States showed prexasertib has both antitumor activity and an acceptable safety profile. A new clinical trial extends these findings by looking at how the drug performs in a new demographic of patients – namely, Japanese patients with advanced solid tumors. Prexasertib is a novel inhibitor of the protein checkpoint kinase 1 – a serine/threonine kinase that promotes DNA repair, controls initiation of DNA replication, and coordinates mitosis. Blocking the protein’s activity prevents cells from resolving replication stress and/or repairing DNA double-strand breaks, which leads to apoptosis. Inhibitors of checkpoint kinase 1 can augment the efficacy of DNA‐damaging chemotherapeutics, but they’re also being evaluated as single-agent therapies..."

The rest of the transcript, along with a link to the research itself, is available on the resource itself.

Subject:
Applied Science
Health, Medicine and Nursing
Material Type:
Diagram/Illustration
Reading
Provider:
Research Square
Provider Set:
Video Bytes
Date Added:
09/20/2019
Stable red blood cell concentrations seen in astronauts on long space missions
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This resource is a video abstract of a research paper created by Research Square on behalf of its authors. It provides a synopsis that's easy to understand, and can be used to introduce the topics it covers to students, researchers, and the general public. The video's transcript is also provided in full, with a portion provided below for preview:

"Radiation, weightlessness, and altered nutrition are just a few of the harsh conditions astronauts face while in space. Though blood parameters _do_ change during space flight, a new study published in the journal, _BMC Hematology_,_ _suggests crews on long missions may not experience persistent anemia, as once thought. Environmental conditions in space are undoubtedly different from those on Earth, and can, therefore, impact a number of the body’s systems including vision, immunity, and the musculoskeletal system. It is well understood that, upon entering microgravity, fluids rapidly shift toward the head -- evident by the so-called ‘puffy-face syndrome’ seen in many astronauts. It has been assumed that during long-duration space missions, this is accompanied by a state of anemia – a condition that occurs when the body fails to produce a sufficient levels of oxygen-carrying red blood cells and can result in weakness and fatigue..."

The rest of the transcript, along with a link to the research itself, is available on the resource itself.

Subject:
Applied Science
Health, Medicine and Nursing
Life Science
Nutrition
Material Type:
Diagram/Illustration
Reading
Provider:
Research Square
Provider Set:
Video Bytes
Date Added:
11/21/2020
Viral infection of hematopoietic stem cells
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CC BY
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This resource is a video abstract of a research paper created by Research Square on behalf of its authors. It provides a synopsis that's easy to understand, and can be used to introduce the topics it covers to students, researchers, and the general public. The video's transcript is also provided in full, with a portion provided below for preview:

"Hematopoietic stem cells (HSCs) are critical for maintaining healthy blood and immune cell populations. They’re also valuable resources and targets for medical treatments, such as HSC transplantation and gene therapy. However, these blood cell precursors are susceptible to viral infection, which can cause blood disorders and limit the efficacy of HSC-based therapies. In fact, viral infection is a leading cause of complications and death among HSC transplant recipients. For example, latent cytomegalovirus can become reactivated after transplantation leading to immunosuppression, pneumonia, encephalitis, and graft failure. HSC transplantation also reduces the numbers of T cells that are specifically cytotoxic toward the mononucleosis- inducing Epstein–Barr virus. Furthermore, recipients of HSC transplants are more susceptible to infection by SARS-CoV-2, the cause of the COVID-19 pandemic..."

The rest of the transcript, along with a link to the research itself, is available on the resource itself.

Subject:
Biology
Life Science
Material Type:
Diagram/Illustration
Reading
Provider:
Research Square
Provider Set:
Video Bytes
Date Added:
05/15/2023