Gene therapy comes of age

CE Dunbar, KA High, JK Joung, DB Kohn, K Ozawa… - Science, 2018 - science.org
BACKGROUND Nearly five decades ago, visionary scientists hypothesized that genetic
modification by exogenous DNA might be an effective treatment for inherited human …

CAR T cells: continuation in a revolution of immunotherapy

AK Singh, JP McGuirk - The Lancet Oncology, 2020 - thelancet.com
The recent clinical successes of immunotherapy, as a result of a broader and more profound
understanding of cancer immunobiology, and the leverage of this knowledge to effectively …

Viral vectors for gene therapy: translational and clinical outlook

MA Kotterman, TW Chalberg… - Annual review of …, 2015 - annualreviews.org
In a range of human trials, viral vectors have emerged as safe and effective delivery vehicles
for clinical gene therapy, particularly for monogenic recessive disorders, but there has also …

Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure.

PL Felgner, TR Gadek, M Holm… - Proceedings of the …, 1987 - National Acad Sciences
A DNA-transfection protocol has been developed that makes use of a synthetic cationic lipid,
N-[1-(2, 3-dioleyloxy) propyl]-N, N, N-trimethylammonium chloride (DOTMA). Small …

Bcl-2 gene promotes haemopoietic cell survival and cooperates with c-myc to immortalize pre-B cells

DL Vaux, S Cory, JM Adams - Nature, 1988 - nature.com
A common feature of follicular lymphoma, the most prevalent haematological malignancy in
humans, is a chromosome translocation (t (14; 18)) that has coupled the immunoglobulin …

T Lymphocyte-Directed Gene Therapy for ADA SCID: Initial Trial Results After 4 Years

RM Blaese, KW Culver, AD Miller, CS Carter, T Fleisher… - Science, 1995 - science.org
In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine
deaminase (ADA) gene into the T cells of two children with severe combined …

Ligand-targeted particulate nanomedicines undergoing clinical evaluation: current status

R van der Meel, LJC Vehmeijer, RJ Kok… - Advanced drug delivery …, 2013 - Elsevier
Since the introduction of Doxil® on the market nearly 20 years ago, a number of
nanomedicines have become part of treatment regimens in the clinic. With the exception of …

[HTML][HTML] Gene therapy-promises, problems and prospects

IM Verma, N Somia - Nature, 1997 - nature.com
The concept behind gene therapy is simple—by delivering corrective genetic material to the
cells of a patient the symptoms of disease can be alleviated. But seven years after the first …

Advanced mammalian gene transfer: high titre retroviral vectors with multiple drug selection markers and a complementary helper-free packaging cell line

JP Morgenstern, H Land - Nucleic acids research, 1990 - academic.oup.com
We report the development of an advanced system for transfer and expression of exogenous
genes in mammalian cells based on Moloney murine leukemia virus (Mo MuLV). Extensive …

[HTML][HTML] The T4 gene encodes the AIDS virus receptor and is expressed in the immune system and the brain

PJ Maddon, AG Dalgleish, JS McDougal, PR Clapham… - Cell, 1986 - cell.com
The isolation of clones encoding the human surface protein T4, and the expression of the T4
gene in new cellular environments, have enabled us to examine the role of this protein in the …