| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
AAV vector development, back to the future
|
Suarez-Amaran, Lester
|
|
|
33 |
5 |
p. 1903-1936
|
artikel
|
| 2 |
ABI and generative biology: A new paradigm for gene therapy, genome engineering, and engineered cell therapy
|
Woolfson, Adrian
|
|
|
33 |
5 |
p. 1881-1885
|
artikel
|
| 3 |
Addressing barriers to clinical translation of extracellular vesicle therapeutics
|
Jay, Steven M.
|
|
|
33 |
5 |
p. 1879-1880
|
artikel
|
| 4 |
Advances in AAV capsid engineering: Integrating rational design, directed evolution and machine learning
|
Nisanov, Alan M.
|
|
|
33 |
5 |
p. 1937-1945
|
artikel
|
| 5 |
A fantastically encapsulated idea
|
Campbell, Matthew
|
|
|
33 |
5 |
p. 1864
|
artikel
|
| 6 |
A synthetic opsin restores vision in patients with severe retinal degeneration
|
Mohanty, Samarendra K.
|
|
|
33 |
5 |
p. 2279-2290
|
artikel
|
| 7 |
Celebrating 25 years of Molecular Therapy
|
Glorioso, Joseph C.
|
|
|
33 |
5 |
p. 1861
|
artikel
|
| 8 |
Current and future treatments for sickle cell disease: From hematopoietic stem cell transplantation to in vivo gene therapy
|
Ball, Julia
|
|
|
33 |
5 |
p. 2172-2191
|
artikel
|
| 9 |
Current trends in gene therapy to treat inherited disorders of the brain
|
Matuszek, Zaneta
|
|
|
33 |
5 |
p. 1988-2014
|
artikel
|
| 10 |
Discovery and preclinical development of a potent epigenic editor targeting PCSK9 to lower LDL cholesterol
|
Xiong, Qiang
|
|
|
33 |
5 |
p. 1874-1875
|
artikel
|
| 11 |
Draining the brain: Gene therapy reverses brain edema in mice with the leukodystrophy MLC
|
Min, Rogier
|
|
|
33 |
5 |
p. 1872-1873
|
artikel
|
| 12 |
Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy
|
Williams, David A.
|
|
|
33 |
5 |
p. 2141-2153
|
artikel
|
| 13 |
Four decades of adenovirus gene transfer vectors: History and current use
|
Hackett, Neil R.
|
|
|
33 |
5 |
p. 2192-2204
|
artikel
|
| 14 |
From concept to cure: The evolution of CAR-T cell therapy
|
Patel, Kisha K.
|
|
|
33 |
5 |
p. 2123-2140
|
artikel
|
| 15 |
Gene regulation technologies for gene and cell therapy
|
Butterfield, Gabriel L.
|
|
|
33 |
5 |
p. 2104-2122
|
artikel
|
| 16 |
Gene therapy for hemophilia – From basic science to first approvals of “one-and-done” therapies
|
Herzog, Roland W.
|
|
|
33 |
5 |
p. 2015-2034
|
artikel
|
| 17 |
Gene therapy then and now: A look back at changes in the field over the past 25 years
|
Wang, Dan
|
|
|
33 |
5 |
p. 1889-1902
|
artikel
|
| 18 |
Genome editing strategies for targeted correction of β-globin mutation in sickle cell disease: From bench to bedside
|
Butt, Henna
|
|
|
33 |
5 |
p. 2154-2171
|
artikel
|
| 19 |
GMP manufacturing of cell and gene therapy products: Challenges, opportunities, and pathways forward
|
Fury, Brian
|
|
|
33 |
5 |
p. 1886-1888
|
artikel
|
| 20 |
High-dimensional temporal mapping of CAR T cells reveals phenotypic and functional remodeling during manufacturing
|
Cadinanos-Garai, Amaia
|
|
|
33 |
5 |
p. 2291-2309
|
artikel
|
| 21 |
How to democratize cell and gene therapy: A global approach
|
Rouce, Rayne H.
|
|
|
33 |
5 |
p. 2082-2090
|
artikel
|
| 22 |
Immuno-oncology recapitulates ontogeny: Modern cell and gene therapy for cancer
|
Carleton, Neil
|
|
|
33 |
5 |
p. 2229-2237
|
artikel
|
| 23 |
In this issue
|
|
|
|
33 |
5 |
p. 1865
|
artikel
|
| 24 |
Machine learning approaches enable the discovery of therapeutics across domains
|
Chhibbar, Prabal
|
|
|
33 |
5 |
p. 2269-2278
|
artikel
|
| 25 |
Molecular Therapy Family Highlights
|
|
|
|
33 |
5 |
p. 1866
|
artikel
|
| 26 |
Next-generation replication-defective HSV vectors for delivery of large DNA payloads
|
Ingusci, Selene
|
|
|
33 |
5 |
p. 2205-2216
|
artikel
|
| 27 |
Oncolytic viruses as cancer therapeutics: From mechanistic insights to clinical translation
|
Alwithenani, Akram
|
|
|
33 |
5 |
p. 2217-2228
|
artikel
|
| 28 |
Perspectives and open questions in vision restoration with vMCO-010
|
Lindner, Moritz
|
|
|
33 |
5 |
p. 1867
|
artikel
|
| 29 |
Prenatal treatment of spinal muscular atrophy
|
Servais, Laurent
|
|
|
33 |
5 |
p. 1862-1863
|
artikel
|
| 30 |
Progress in skin gene therapy: From the inside and out
|
Osborn, Mark J.
|
|
|
33 |
5 |
p. 2065-2081
|
artikel
|
| 31 |
Recent developments in gene therapy for Parkinson’s disease
|
Szunyogh, Sandor
|
|
|
33 |
5 |
p. 2052-2064
|
artikel
|
| 32 |
Recent progress and future challenges in structure-based protein-protein interaction prediction
|
Yuan, Rongqing
|
|
|
33 |
5 |
p. 2252-2268
|
artikel
|
| 33 |
Redefining quality in cell and gene therapies: Lessons from implementing electronic QMS in academic cGMP facility
|
Wu, Xia
|
|
|
33 |
5 |
p. 2091-2103
|
artikel
|
| 34 |
Response: Promise and open questions of optogenetic vision restoration by MCO
|
Mohanty, Samarendra K.
|
|
|
33 |
5 |
p. 1868
|
artikel
|
| 35 |
Response to: Safety and efficacy considerations of HSC-based gene therapy for RAG1-deficient SCID
|
Montini, Eugenio
|
|
|
33 |
5 |
p. 1871
|
artikel
|
| 36 |
Safety and efficacy of gene therapy for RAG1-deficient SCID
|
Staal, Frank J.T.
|
|
|
33 |
5 |
p. 1869-1870
|
artikel
|
| 37 |
Small RNAs as therapeutic agents: From catalytic motifs to regulatory pathways
|
Rossi, John J.
|
|
|
33 |
5 |
p. 2238-2242
|
artikel
|
| 38 |
The curious case of AAV immunology
|
Keeler, Allison M.
|
|
|
33 |
5 |
p. 1946-1965
|
artikel
|
| 39 |
The deLIVERed promises of gene therapy: Past, present, and future of liver-directed gene therapy
|
Puzzo, Francesco
|
|
|
33 |
5 |
p. 1966-1987
|
artikel
|
| 40 |
The past, present, and future of RNA vaccines
|
Tregoning, John S.
|
|
|
33 |
5 |
p. 1876-1878
|
artikel
|
| 41 |
Therapeutic application of extracellular vesicles in human diseases
|
Nunes, Allancer
|
|
|
33 |
5 |
p. 2243-2251
|
artikel
|
| 42 |
The road toward AAV-mediated gene therapy of Duchenne muscular dystrophy
|
Bengtsson, Niclas E.
|
|
|
33 |
5 |
p. 2035-2051
|
artikel
|
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