2012 DNA Therapies 
Drug Target Review
JANUARY 8, 2024
Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. a clinical-stage private biopharma company developing therapies for neurological and psychiatric diseases.
PLOS: DNA Science
OCTOBER 12, 2023
In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.
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Drug Target Review
OCTOBER 26, 2023
Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union. Molecular Therapy 20 , 1831-1832 (2012). Nature 578 , 229-236 (2020).
Drug Target Review
NOVEMBER 6, 2023
2 It is this complexity that necessitates powerful, targeted combination therapies. 4, 5 More recently, new combination regimens have emerged that incorporate targeted therapies to treat a variety of blood cancers, including multiple myeloma (MM), chronic lymphocytic leukaemia (CLL) and acute myelogenous leukaemia (AML).
Drug Target Review
OCTOBER 4, 2023
2 In response, DNA-damaging agents that could target the entire cell cycle received renewed attention as ADC payloads. Dual payload ADCs As effective as therapies have been in treating solid and haematological cancers, tumour heterogeneity and resistance remain major clinical challenges. Fu Y, Ho M. 1 , 33–43 (2018).
Codon
JUNE 11, 2024
And unlike traditional DNA sequencers, which parse genetic material by breaking it up into fragments and interpreting it chunk-by-chunk, a nanopore device unspools a long strand of DNA and reads it all at once. A scientist can isolate DNA and load up a flow cell in fifteen minutes. Nanopore devices work incredibly fast.
Drug Target Review
AUGUST 9, 2023
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.
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