Results 351 to 360 of about 12,497,887 (407)
Programmable Single-Stranded Circular Antisense Oligonucleotides for Multitarget Gene Therapy. [PDF]
JACS AuPan Y +11 more
europepmc +1 more source
Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1.
JAMA Netw OpenRopars J +18 more
europepmc +1 more source
Some of the next articles are maybe not open access.
Related searches:
Related searches:
New England Journal of Medicine, 2019
Gene therapy medicinal products (GTMPs) are one of the most promising biopharmaceuticals, which are beginning to show encouraging results. The broad clinical research activity has been addressed mainly to cancer, primarily to those cancers that do not respond well to conventional treatment.
del Pozo-Rodriguez A. +6 more
openaire +5 more sources
Gene therapy medicinal products (GTMPs) are one of the most promising biopharmaceuticals, which are beginning to show encouraging results. The broad clinical research activity has been addressed mainly to cancer, primarily to those cancers that do not respond well to conventional treatment.
del Pozo-Rodriguez A. +6 more
openaire +5 more sources
Exosome-mediated delivery of gene vectors for gene therapy.
Nanoscale, 2020Gene vectors are nucleic acids that carry genetic materials or gene editing devices into cells to exert the sustained production of therapeutic proteins or to correct erroneous genes of the cells.
Li Duan +7 more
semanticscholar +1 more source
Gene therapy for leukodystrophies
Human Molecular Genetics, 2011Leukodystrophies (LDs) refer to a group on inherited diseases in which molecular abnormalities of glial cells are responsible for exclusive or predominant defects in myelin formation and/or maintenance within the central and, sometimes, the peripheral nervous system.
Biffi A., Aubourg P., Cartier N.
openaire +3 more sources
Journal of Cellular Physiology, 2020
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR‐associated enzyme (Cas) is a naturally occurring genome editing tool adopted from the prokaryotic adaptive immune defense system.
Baohong Zhang
semanticscholar +1 more source
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR‐associated enzyme (Cas) is a naturally occurring genome editing tool adopted from the prokaryotic adaptive immune defense system.
Baohong Zhang
semanticscholar +1 more source
Fetal Diagnosis and Therapy, 1987
Severe genetic disorders are potentially correctable by the addition of a normal gene into tissues. Although the technical problems involving integration, stable expression, and insertional damage to the treated cell are not yet fully solved, enough scientific progress has already been made to consider somatic cell gene therapy acceptable from both the
A, Drugan, O J, Miller, M I, Evans
openaire +2 more sources
Severe genetic disorders are potentially correctable by the addition of a normal gene into tissues. Although the technical problems involving integration, stable expression, and insertional damage to the treated cell are not yet fully solved, enough scientific progress has already been made to consider somatic cell gene therapy acceptable from both the
A, Drugan, O J, Miller, M I, Evans
openaire +2 more sources
Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy
Human Gene Therapy, 2020N/A.
James M. Wilson, T. Flotte
semanticscholar +1 more source