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Landing therapeutic genes safely in the human genome

A team of researchers at Harvard's Wyss Institute and ETH Zurich have developed a computational approach to identify genomic safe harbors (GSHs) with high potential for safe insertion of therapeutic genes. The study validated two GSH sites in adoptive T cell therapies and in vivo gene therapies for skin diseases.

SourceWyss Institute for Biologically Inspired Engineering at Harvard·JournalCell Reports Methods·TypeExperimental study·DateJan 24, 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.

New study reveals how epithelial cells in the body naturally eliminate “precancerous” ones

Researchers have identified a novel immune-like mechanism by which healthy epithelial cells recognize and eliminate precancerous cells through a MHC class I-LILRB3 interaction. This process generates mechanical force to extrude the precancerous cells from the body, offering new hope for cancer prevention and treatment.

SourceWaseda University·JournalNature Immunology·TypeExperimental study·DateDec 15, 2021

Building better tools for biomanufacturing

A team of researchers has developed a Dynamic Sampling Platform to analyze cells in real-time, overcoming the time-consuming and expensive process of biomanufacturing. The platform provides insight into cell behavior and biochemical information needed for process control, potentially lowering the cost of cell therapies.

SourceGeorgia Institute of Technology·JournalLab on a Chip·TypeExperimental study·DateDec 2, 2021

Heart repair and regeneration after a heart attack — a review

Recent clinical trials showed promising results with cardiosphere-derived cells, improving heart parameters in patients with Duchenne muscular dystrophy. Researchers investigate using cell-derived products like exosomes to boost endogenous repair pathways, while aiming to reverse cardiomyocytes' proliferation limitations.

SourceUniversity of Alabama at Birmingham·JournalJournal of the American College of Cardiology·TypeLiterature review·DateNov 29, 2021

Personalized T cell immunotherapy for pediatric brain tumors is one step closer to becoming a reality

Researchers at Children's National Hospital have successfully developed a personalized T cell immunotherapy that targets and kills unique proteins in individual tumor cells. This approach, combining genetic sequencing and protein identification, offers a promising treatment option for children with hard-to-treat brain tumors.

SourceChildren's National Hospital·JournalNature Communications·DateNov 18, 2021

Fasting-mimicking diet is safe, may modulate metabolism and boost antitumor immunity in cancer patients

A fasting-mimicking diet was found to be safe and biologically active in cancer patients, with potential benefits for modulation of metabolism and enhancement of antitumor immunity. The study demonstrated significant reductions in blood glucose and growth factor concentrations, as well as enhancements in intratumor T-cell infiltration.

SourceAmerican Association for Cancer Research·JournalCancer Discovery·DateNov 17, 2021

Study shows that the heart’s own immune cells hold potential for the treatment of heart failure

A new study led by University of Minnesota Medical School researcher Xavier Revelo found that macrophages play a role in protecting the heart after injury. The research team discovered a large increase in cardiac macrophages early in response to a cardiac injury similar to high blood pressure.

SourceUniversity of Minnesota Medical School·JournalCirculation Research·TypeExperimental study·DateNov 12, 2021

Differences in T cells’ functional state determine resistance to cancer therapy

Researchers found that certain T cells stop working before entering the tumor due to changes in gene expression, making ICB therapies less effective. Combining ICB with other forms of immunotherapy targeting different aspects of T cell function may improve response rates for non-small cell lung cancer patients.

SourceMassachusetts Institute of Technology Department of Biology·JournalScience Immunology·TypeExperimental study·DateOct 29, 2021

Engineers devise a way to selectively turn on RNA therapies in human cells

Researchers at MIT and Harvard University have developed a way to selectively turn on gene therapies in target cells by detecting specific messenger RNA sequences. This technology can fine-tune gene therapies for applications ranging from regenerative medicine to cancer treatment, potentially reducing side effects and increasing efficacy.

SourceMassachusetts Institute of Technology·JournalNature Biotechnology·DateOct 28, 2021

Potential new treatment for COVID-19 identified

Researchers have discovered a potential new treatment for COVID-19 by targeting the pentose phosphate pathway, which is necessary for SARS-CoV-2 replication. The study found that inhibiting this pathway with benfooxythiamine suppresses viral replication and reduces virus production.

SourceUniversity of Kent·JournalMetabolites·TypeExperimental study·DateOct 28, 2021

A new treatment for glaucoma?

Researchers developed a new protein treatment that prevents glaucoma from forming in mice and reduces pressure in the eyes. The study provides new targets for therapies and aims to develop an injectable treatment for patients.

SourceNorthwestern University·JournalNature Communications·DateOct 18, 2021

A Chinese medical journal review sheds light on T-cell responses in respiratory diseases

Researchers comprehensively review T-cell responses to respiratory viral infections and chronic obstructive pulmonary disease (COPD), highlighting key characteristics of peptide-reactive T-cells. The review aims to improve understanding of the underlying mechanisms, leading to more effective immune protection and treatment methods.

SourceCactus Communications·JournalChinese Medical Journal·TypeLiterature review·DateSep 30, 2021

New study suggests that aquaporin could be key to repairing corneal defects

A new study provides evidence supporting the involvement of aquaporins in corneal cell proliferation and nerve regeneration, suggesting AQP5 induction as a potential therapy to accelerate corneal defect resurfacing. The study found that AQP5 deficiency can slow down corneal epithelial repair, but its specific mechanism remained unclear.

SourceElsevier·JournalAmerican Journal Of Pathology·TypeExperimental study·DateSep 29, 2021

Mount Sinai scientists show that a novel therapy could be effective against pediatric leukemia

Researchers at Mount Sinai have developed a novel therapy called MS67 that effectively fights acute myeloid leukemia with mixed lineage leukemia rearrangement. The therapy degrades the WDR5 protein, which drives the proliferation of this type of leukemia and other cancers such as pancreatic cancer.

SourceThe Mount Sinai Hospital / Mount Sinai School of Medicine·JournalScience Translational Medicine·DateSep 29, 2021

Early signs of efficacy of new targeted agents and immunotherapies reported for multiple cancers at ESMO Congress 2021

New targeted therapies are being developed to target genetic alterations in cancer cells, such as the ARID1A mutation found in 10-50% of solid tumours. Early clinical trials suggest that these agents may be effective in treating multiple cancers, including breast, ovarian, and gastric cancer.

SourceEuropean Society for Medical Oncology·JournalAnnals of Oncology·DateSep 24, 2021

Hopkins Med news update

Researchers discovered that new mutations in the BRAF gene can lead to gliomas growing back after treatment, suggesting personalized approaches to therapy. The study also explored potential impacts of COVID-19 vaccines on menstruation.

SourceJohns Hopkins Medicine·JournalClinical Cancer Research·DateSep 23, 2021

Special transcription factor and its target genes as an important approach for therapy at rare leukemia type identified

A study by MedUni Wien researchers has discovered that the transcription factor BATF3 and its target genes play a crucial role in the growth of tumour cells in anaplastic large cell lymphoma. The findings suggest that targeting the IL-2R system could be an effective therapeutic approach, with promising results in animal models.

SourceMedical University of Vienna·JournalNature Communications·DateSep 22, 2021

Discovery of a universal system for transporting nucleic acids into cells

A team of researchers from IOCB Prague has discovered a new type of nanoparticles capable of safely transporting various types of nucleic acids used for therapeutic purposes into cells. The universal nature of their system sets it apart from existing solutions, allowing for efficient transport of mRNA and other RNA molecules into cells.

Promising autologous cell candidates identified as potential delivery vehicles for Hemophilia A treatment

A recent study found that amniotic fluid-derived, neonatal, and adult cells can be used to deliver long-lasting Factor VIII protein for Hemophilia A treatment. The researchers identified cells from umbilical cord tissue as the most promising candidates, which yielded high levels of Factor VIII mRNA and blood clotting activity.

SourceAtrium Health Wake Forest Baptist·JournalFrontiers in Cell and Developmental Biology·DateSep 17, 2021

Research provides new insight into fundamental workings of the immune system in response to therapy to treat skin cancer

Researchers at the University of Birmingham discovered that T cells send messages from five specific genes in their immune response to drugs given to treat skin cancer. This finding suggests that an optimal level of stimulation is required for a strong immune response, and blocking certain immune brakes may re-awaken dormant T cells.

SourceUniversity of Birmingham·JournalImmunity·DateSep 16, 2021

Adoptive transfer of tumor-infiltrating lymphocytes may be less effective in patients with pretreated metastatic melanoma

Patients with relapsed metastatic melanoma who received prior anti-PD-1 therapies had a lower response rate to adoptive cell transfer of tumor-infiltrating lymphocytes (ACT-TIL). ACT-TIL was less effective in these patients compared to those who had never received anti-PD-1 therapy. The study found that the objective response rate and ...

SourceAmerican Association for Cancer Research·JournalClinical Cancer Research·DateAug 19, 2021