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Lab grown, self-sustainable muscle cells repair muscle injury and disease, mouse study shows

Scientists at Johns Hopkins Medicine have successfully cultivated human muscle stem cells capable of renewing themselves and repairing muscle tissue damage in mice. The self-renewing stem cells were created by reprogramming laboratory-grown human skin cells, which then differentiated into specific cell types using a nutrient-rich broth.

SourceJohns Hopkins Medicine·JournalCell Stem Cell·DateApr 20, 2022

A donation that could help cure blood cancer

A study published in Leukemia found that cord blood transplantation (CBT) was more effective than matched related donor transplantation (MRDT) for patients with refractory and relapsed acute myeloid leukemia (R/R AML). The study compared the survival rates of 1,738 CBT-treated patients with those of 713 MRDT-treated patients, revealing...

SourceOsaka University·JournalLeukemia·TypeObservational study·DateJan 5, 2022

Penn Medicine awarded $14 million NIH grant to apply CAR T immunotherapies to match more patients in need of kidney transplants

A clinical trial will harness synthetic chimeric antigen receptor (CAR) T cells to deplete immune B cells and plasma cells producing donor-specific antibodies, aiming to achieve a compatible kidney match for patients with pre-existing antibodies. The NIH-funded study, led by Penn Medicine, intends to begin enrolling patients in 2022.

Surviving ‘butterfly disease’: long-term success of a new gene and stem-cell therapy for EB verified

A new gene and stem-cell therapy has been proven to be effective in treating Epidermolysis Bullosa (EB), a genetic skin disorder, without any side effects. The treatment, which involves transplanting genetically modified skin cells, has resulted in stable results after five years, with the patient now 13 years old.

SourceUniversity of Münster·JournalNew England Journal of Medicine·DateDec 10, 2021

How alike are the cancer cells from a single patient?

A new study by USC researchers uses a genetic technology to analyze gene expression signatures of individual cancer cells from patients with leukemia. The findings show that cancer cells with distinct gene expression profiles tend to grow in different organs, while those with specific genes are more resistant to chemotherapy.

SourceKeck School of Medicine of USC·JournalNature Communications·TypeExperimental study·DateNov 11, 2021

New technique may lead to safer stem cell transplants

Researchers have developed a new method of stem cell transplantation that does not require chemotherapy or radiation. The approach uses immunotherapeutic drugs to eliminate cancer cells and prevent immune rejection. This technique has the potential to be curative for blood cancers and treat other diseases like sickle cell anemia.

SourceWashU Medicine·JournalJournal of Clinical Investigation·DateNov 11, 2021

A decade after gene therapy, children born with deadly immune disorder remain healthy

Researchers report that 90% of patients who received gene therapy between 2009 and 2012 remain disease-free, with significant immune system differences observed among those treated. The therapy is most effective in younger children, but further work is needed to achieve high levels of gene correction in all patients.

SourceUniversity of California - Los Angeles Health Sciences·JournalBlood·TypeRandomized controlled/clinical trial·DateOct 15, 2021

Engineered cells successfully treat cardiovascular and pulmonary disease

Scientists at UCSF have shown that gene-edited cellular therapeutics can effectively treat major diseases such as peripheral artery disease, chronic obstructive pulmonary disease, and heart failure. The study used specially engineered induced pluripotent stem cells called HIP cells to evade the immune system.

SourceUniversity of California - San Francisco·JournalProceedings of the National Academy of Sciences·DateJul 6, 2021

Toward safer stem cell transplants

Researchers are developing new conditioning therapies with fewer side effects, targeting specific proteins on hematopoietic stem cells to make room for healthy ones. This could lead to more patients being able to safely undergo stem cell treatments, reducing complications and improving outcomes.

SourceAmerican Chemical Society·JournalChemical & Engineering News·DateApr 21, 2021

Formula predicts ideal dose of stem cells to cure HIV

A mathematical model developed by researchers predicts that autologous gene-edited stem cell transplants could achieve viral control after antiretroviral therapy withdrawal. The conditions required for this include a sufficient dose of edited stem cells and allowing them to repopulate the blood before ART is stopped.

SourceeLife·DateJan 12, 2021

A 'cell-less' therapy may regenerate heart tissue without cell transplant risks

Researchers developed an 'cell-less' therapy that accelerates recovery from heart attack in pigs by injecting tiny membrane-bound sacs containing proteins and DNA into the heart. This approach avoids safety concerns associated with whole-cell transplants, which often fail to engraft and can cause severe health issues.

SourceAmerican Association for the Advancement of Science (AAAS)·JournalScience Translational Medicine·DateSep 16, 2020

The Lancet HIV: Study suggests a second patient has been cured of HIV

A study of a second HIV patient to undergo successful stem cell transplantation from donors with a HIV-resistant gene finds that there was no active viral infection in the patient's blood 30 months after they stopped anti-retroviral therapy. The patient had a healthy CD4 cell count, suggesting they have recovered well from the transplant.

SourceThe Lancet·JournalThe Lancet HIV·DateMar 10, 2020