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CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. CRISPR also addresses another limitation in CAR T therapies related to the insufficient quantity and poor quality of autologous T cells.

Disease 246
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A Molecular Portrait of ALS and FTD

Drug Target Review

The specific proteins found in these aggregates can vary, but approximately 95 percent of ALS patients and 50 percent of FTD patients develop aggregates containing the TDP-43 protein (short for tar DNA binding protein 43). 14 TDP-43 is a DNA and RNA binding protein that regulates the expression and splicing of several target transcripts.

RNA 98
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Making Cells Young

Codon

Most cells (excluding T-cells and B cells after V(D)J recombination) in our body contain the same DNA but appear and behave in distinct ways: A neuron looks and acts very differently from a hepatocyte. While DNA is relatively stable , the epigenome is not; it has to orchestrate changes in cell state, cell type, and more.

DNA 59
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Beyond Steel Tanks

Codon

Water accounts for 70 percent of a bacterium by mass; the other 30 percent includes everything else: proteins, RNA, DNA, lipids, and so on. Experiments around using genetically modified organisms to produce proteins have been taking place since the earliest days of the recombinant DNA revolution.

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Precision Medicine in the Genomic Era

DrugBank

Their use, as well as their integration into other types of data, has enabled the development of more precise therapies. In contrast, non-coding mutations have effects on gene expression, transcript stability, and the physical state of the DNA itself (e.g., 13 , 1–27 (2012). & Barbadilla, A.  Olson, M.

Disease 89
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Codon Digest: Hackathon Prize Winners

Codon

Since the last Codon Digest, I’ve published: Reasons to Be Grateful for Biotechnology (with Avadhoot Jadhav) AAV Foundations (Part I) An overview of AAV-based gene therapies, how they get made, and where they go wrong. DNA sequences are designed on a computer, and it takes a dozen or more clicks to change a single nucleotide.

DNA 52
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Codon Digest: Hackathon Prize Winners

Codon

Since the last Codon Digest, I’ve published: Reasons to Be Grateful for Biotechnology (with Avadhoot Jadhav) AAV Foundations (Part I) An overview of AAV-based gene therapies, how they get made, and where they go wrong. DNA sequences are designed on a computer, and it takes a dozen or more clicks to change a single nucleotide.

DNA 52