Featured
-
-
Article |
CRISPR/Cas9-based genome editing for the modification of multiple duplications that cause Duchenne muscular dystrophy
- Dan-Ni Wang
- , Zhi-Qiang Wang
- & Ning Wang
-
Article
| Open AccessHIV- 1 lentivirus tethering to the genome is associated with transcription factor binding sites found in genes that favour virus survival
- Saqlain Suleman
- , Annette Payne
- & Michael Themis
-
Article |
Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec –routine care evidence
- Sharon Aharoni
- , Jacob Bistritzer
- & Yoram Nevo
-
Article |
Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy
- Aoife Reilly
- , Marc-Olivier Deguise
- & Rashmi Kothary
-
Comment |
Micro-dystrophin gene constructs for repairing heart and muscle function in rats: the smaller is enough?
- Petra Lujza Szabo
- & Attila Kiss
-
Article |
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene
- Yuan Liu
- , Jeremy D. Eastwood
- & Hong Yu
-
Review Article
| Open AccessEffective viral-mediated lung gene therapy: is airway surface preparation necessary?
- Alexandra McCarron
- , Patricia Cmielewski
- & Martin Donnelley
-
-
Brief Communication |
Prevalence of AAV2.5 neutralizing antibodies in synovial fluid and serum of patients with osteoarthritis
- Temilola Y. Abdul
- , Gresin P. Hawse
- & Rodolfo E. De La Vega
-
Comment |
Friend or Foe(tal): challenges in development of a large animal model for pre-clinical fetal gene therapy
- Richard S. Finkel
- & Christian L. Lorson
-
-
Article |
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury
- Yu Zhai
- , Shi-Yang Ye
- & Yuan-Guo Zhou
-
Article |
Exosomal microRNA-133b-3p from bone marrow mesenchymal stem cells inhibits angiogenesis and oxidative stress via FBN1 repression in diabetic retinopathy
- Gaohua Liang
- , Zhiliang Qin
- & Wenhao Ma
-
Brief Communication |
Targeting mAKAPβ expression as a therapeutic approach for ischemic cardiomyopathy
- Eliana C. Martinez
- , Jinliang Li
- & Michael S. Kapiloff
-
Article |
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model
- Audrey Bourdon
- , Virginie François
- & Caroline Le Guiner
-
Article
| Open AccessAntisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1
- Siham Ait Benichou
- , Dominic Jauvin
- & Jack Puymirat
-
-
Perspective |
Equitable access to cell and gene therapies in South Africa: opportunities and hurdles
- Candice Laverne Hendricks
- , Marco Alessandrini
- & Michael Sean Pepper
-
Article |
Development of a cGMP-compliant process to manufacture donor-derived, CD45RA-depleted memory CD19-CAR T cells
- Young-In Kim-Hoehamer
- , Janice M. Riberdy
- & Sheng Zhou
-
Article |
Upper motor neurons are a target for gene therapy and UCHL1 is necessary and sufficient to improve cellular integrity of diseased upper motor neurons
- Barış Genç
- , Javier H. Jara
- & P. Hande Özdinler
-
Article |
Outer retinal transduction by AAV2-7m8 following intravitreal injection in a sheep model of CNGA3 achromatopsia
- M. Ross
- , A. Obolensky
- & R. Ofri
-
Article |
Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets
- Kelly A. Rich
- , Christopher G. Wier
- & Stephen J. Kolb
-
Article
| Open AccessDystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy
- Vahid Farrokhi
- , Jason Walsh
- & Hendrik Neubert
-
Article
| Open AccessLong-term safety and efficacy of intramyocardial adenovirus-mediated VEGF-DΔNΔC gene therapy eight-year follow-up of phase I KAT301 study
- Aleksi J. Leikas
- , Iiro Hassinen
- & Juha E. K. Hartikainen
-
Article |
Injection pressure levels for creating blebs during subretinal gene therapy
- Brittni A. Scruggs
- , Huber Martins Vasconcelos Jr.
- & Andreas K. Lauer
-
Article
| Open AccessRs1h−/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation
- Yong Zeng
- , Haohua Qian
- & Paul A. Sieving
-
Article |
E3 ubiquitin ligase SMURF2 prevents colorectal cancer by reducing the stability of the YY1 protein and inhibiting the SENP1/c-myc axis
- Qianfu Gao
- , Shanchao Wang
- & Zeyan Zhang
-
Article
| Open AccessDevelopment and biological characterization of a clinical gene transfer vector for the treatment of MAK-associated retinitis pigmentosa
- Budd A. Tucker
- , Erin R. Burnight
- & Edwin M. Stone
-
Review Article
| Open AccessTowards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)
- Jennifer E. Adair
- , Lindsay Androski
- & Boro Dropulić
-
Article
| Open AccessUltra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer
- Krystal Sandza
- , Annie Clark
- & Christian Vettermann
-
Article |
CCR1 enhances SUMOylation of DGCR8 by up-regulating ERK phosphorylation to promote spinal nerve ligation-induced neuropathic pain
- Cunxian Shi
- , Jin Jin
- & Zhen Li
-
Article |
Novel design of nucleic acid standards for hydrolysis probe-based PCR with melting analysis
- Anna Baoutina
- & Somanath Bhat
-
Brief Communication |
Suppression of bone metastatic castration-resistant prostate cancer cell growth by a suicide gene delivered by JC polyomavirus-like particles
- Cheng-Huang Shen
- , Mien-Chun Lin
- & Meilin Wang
-
Article
| Open AccessGene therapy for spinal muscular atrophy: the Qatari experience
- Hossamaldein Gaber Ali
- , Khalid Ibrahim
- & Tawfeg Ben-Omran
-
Perspective
| Open AccessPulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo
- Ewa Janosz
- , Miriam Hetzel
- & Thomas Moritz
-
Article |
rAAV-based and intraprostatically delivered miR-34a therapeutics for efficient inhibition of prostate cancer progression
- Jianzhong Ai
- , Jia Li
- & Guangping Gao
-
Review Article
| Open AccessStem cell-based therapy for hirschsprung disease, do we have the guts to treat?
- Ali Fouad Alhawaj
-
Article
| Open AccessOptimisation of a TALE nuclease targeting the HIV co-receptor CCR5 for clinical application
- Lea Isabell Schwarze
- , Dawid Głów
- & Boris Fehse
-
-
Brief Communication
| Open AccessAdvantages of robotic assistance over a manual approach in simulated subretinal injections and its relevance for gene therapy
- Reza Ladha
- , Thijs Meenink
- & Marc D. de Smet
-
Article |
Distinct functions of CAR-T cells possessing a dectin-1 intracellular signaling domain
- Xiao Liang
- , Yong Huang
- & Wei Wang
-
Article |
Membrane-bound MMP-14 protease-activatable adeno-associated viral vectors for gene delivery to pancreatic tumors
- Susan S. Butler
- , Kenjiro Date
- & Junghae Suh
-
Article
| Open AccessAutomated production of CCR5-negative CD4+-T cells in a GMP-compatible, clinical scale for treatment of HIV-positive patients
- Lea Isabell Schwarze
- , Tanja Sonntag
- & Boris Fehse
-
Article
| Open AccessCARAMBA: a first-in-human clinical trial with SLAMF7 CAR-T cells prepared by virus-free Sleeping Beauty gene transfer to treat multiple myeloma
- Sabrina Prommersberger
- , Michael Reiser
- & Zoltán Ivics
-
Review Article
| Open AccessCurrent status and perspective of CAR-T and CAR-NK cell therapy trials in Germany
- Nawid Albinger
- , Jessica Hartmann
- & Evelyn Ullrich
-
Review Article
| Open AccessRetroviral gene therapy in Germany with a view on previous experience and future perspectives
- Michael A. Morgan
- , Melanie Galla
- & Axel Schambach
-
-
Article |
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair
- Colin L. Sweeney
- , Mara Pavel-Dinu
- & Suk See De Ravin
-
Article |
Striatal ΔFosB gene suppression inhibits the development of abnormal involuntary movements induced by L-Dopa in rats
- Goichi Beck
- , Jie Zhang
- & Stella M. Papa
Browse broader subjects
Browse narrower subjects
- Cancer
- Cardiovascular diseases
- Dental diseases
- Endocrine system and metabolic diseases
- Eye diseases
- Gastrointestinal diseases
- Haematological diseases
- Immunological disorders
- Infectious diseases
- Kidney diseases
- Metabolic disorders
- Neurological disorders
- Nutrition disorders
- Oral diseases
- Psychiatric disorders
- Reproductive disorders
- Respiratory tract diseases
- Rheumatic diseases
- Skin diseases
- Trauma
- Urogenital diseases