Remove DNA Remove Immune Response Remove Therapies
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FDA Returns Disappointing News for ALS Stem Cell Therapy

PLOS: DNA Science

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

Therapies 105
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Regulatory Guidance for Oligonucleotide Bioanalysis in Drug Development

Alta Sciences

These interactions may disrupt protein function, trigger immune responses, or contribute to other toxicological risks. to ligands or linkers) may elicit immune responses. An oligonucleotide is a short strand of nucleotides, the building blocks of DNA or RNA, used in genetic research and therapy.

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mRNA Therapeutics: Revolutionizing Treatment Beyond Vaccines

DrugBank

  The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach. This process triggers a robust immune response, enabling the immune system to recognize and attack cancer cells.

Vaccine 97
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Designing a Better Probiotic. CRISPR Hubris?

PLOS: DNA Science

The molecular tools of CRISPR were borrowed and developed from the natural immune response of bacteria to viruses – bacteriophages – that infect them. CRISPR may make sense for replacing a single mutant gene, like the one behind sickle cell disease, for which a CRISPR-based gene therapy was recently approved.

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The immune system’s role in lung cancer risk

Drug Target Review

HLA are the critical molecules that our immune systems use to present peptides to T cells and facilitate recognition and killing in immune responses to pathogens. HLA-II presents peptides to CD4 T cells thought to be important for indirectly helping immune responses and facilitating antibody production.

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Unveiling neoantigen-directed cancer treatment

Drug Target Review

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

Treatment 105
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New insights into the role of viral capsids in gene therapy safety

Drug Target Review

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.