Remove 2012 Remove DNA Remove Immune Response Remove Therapies
article thumbnail

Is Recent Gene Therapy Setback for Duchenne Muscular Dystrophy (DMD) Déjà vu All Over Again?

PLOS: DNA Science

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.

article thumbnail

AI in gene delivery vector discovery and design

Drug Target Review

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 9 In addition, host immune responses further add to the complexity of developing cell-specific AAV capsids for clinical applications.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

The power of combinations in blood cancers

Drug Target Review

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).

article thumbnail

Antibody-drug conjugates payloads: then, now and next

Drug Target Review

2 In response, DNA-damaging agents that could target the entire cell cycle received renewed attention as ADC payloads. More recently, researchers have begun to look at conjugating immunomodulatory agents to antibodies to directly activate an immune response against the tumour.

article thumbnail

Leveraging flow cytometry in drug discovery 

Drug Discovery World

DNA-based dyes, amine-reactive, fixable dyes) as oversaturated staining can falsely lead to over-exclusion of live cells from analysis. . After initial postgraduate studies at Gothenburg University, he continued his academic career at UCSD characterising the innate immune response against pathogens. Future directions .

Drugs 130