Nature Search

Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy

Optimized base editors targeting the exon-7 mutation in SMN2 restore expression of the survival motor neuron (SMN) protein to normal levels, as shown in mice with spinal muscular atrophy and in fibroblasts from patients with this genetic disease.

Christiano R. R. Alves
Leillani L. Ha
Benjamin P. Kleinstiver
Research06 Dec 2023
Nature Biomedical Engineering

Volume: 8, P: 118-131
Selective delivery of herpes virus vectors to experimental brain tumors using RMP-7

Faith H Barnett
Nikolai G Rainov
Xandra O Breakefield
Research22 Oct 1999
Cancer Gene Therapy

Volume: 6, P: 14-20
Enhanced green fluorescent protein fusion proteins of herpes simplex virus type 1 thymidine kinase and cytochrome P450 4B1: Applications for prodrug-activating gene therapy

Sabine Steffens
Susanne Frank
Christof M Kramm
Research15 Jun 2000
Cancer Gene Therapy

Volume: 7, P: 806-812
Reactivated endogenous retroviruses promote protein aggregate spreading

Endogenous retroviruses, or genomic relics of ancient viral infection, have been associated with certain neurodegenerative diseases. Here, Liu et al. report a pathway by which reactivated viral gene products contribute to intercellular protein aggregate spreading.

Shu Liu
Stefanie-Elisabeth Heumüller
Ina M. Vorberg
ResearchOpen Access18 Aug 2023
Nature Communications

Volume: 14, P: 1-19
Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial

A phase 1/2 trial of dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis, administered by bilateral intrathalamic, cisterna magna and intrathecal delivery found a dose-dependent biochemical correction of the disease.

Florian Eichler
Oguz I. Cataltepe
Terence R. Flotte
ResearchOpen Access15 Aug 2025
Nature Medicine

Volume: 31, P: 2927-2935
AAV gene therapy for Tay-Sachs disease

First-in-human combined intrathalamic and intrathecal gene therapy in two patients with Tay-Sachs disease provides early evidence on the safety and feasibility of the approach.

Terence R. Flotte
Oguz Cataltepe
Miguel Sena-Esteves
Research10 Feb 2022
Nature Medicine

Volume: 28, P: 251-259
Multimodal targeted high relaxivity thermosensitive liposome for in vivo imaging

Maayke M. P. Kuijten
M. Hannah Degeling
Bakhos A. Tannous
ResearchOpen Access27 Nov 2015
Scientific Reports

Volume: 5, P: 1-11
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

A divalent siRNA architecture enables sustained silencing of gene expression in deep regions of the brain.

Julia F. Alterman
Bruno M. D. C. Godinho
Anastasia Khvorova
Research02 Aug 2019
Nature Biotechnology

Volume: 37, P: 884-894
Adeno-associated viral vectors for functional intravenous gene transfer throughout the non-human primate brain

Crossing the blood–brain barrier in primates is a major obstacle to gene delivery in the brain. Here an adeno-associated virus variant (AAV.CAP-Mac) is identified and demonstrated for crossing the blood–brain barrier and delivering gene sequences to the brain of different non-human primates species.

Miguel R. Chuapoco
Nicholas C. Flytzanis
Viviana Gradinaru
ResearchOpen Access10 Jul 2023
Nature Nanotechnology

Volume: 18, P: 1241-1251
GDNF gene therapy for alcohol use disorder in male non-human primates

In a preclinical study, the delivery of an AAV-based gene therapy encoding GDNF in the brain prevented the return to alcohol use behaviors in a non-human primate model.

Matthew M. Ford
Brianna E. George
Krystof S. Bankiewicz
Research14 Aug 2023
Nature Medicine

Volume: 29, P: 2030-2040
Retrograde neuronal tracing with a deletion-mutant rabies virus

Ian R Wickersham
Stefan Finke
Edward M Callaway
Research10 Dec 2006
Nature Methods

Volume: 4, P: 47-49
Therapeutic benefit of combining calorie-restricted ketogenic diet and glutamine targeting in late-stage experimental glioblastoma

Purna Mukherjee et al demonstrate that administering a glutamine analogue together with a calorie-restricted ketogenic diet can reduce brain tumour growth and inflammation, while enhancing survival, in two syngeneic orthotopic mouse models.

Purna Mukherjee
Zachary M. Augur
Thomas N. Seyfried
ResearchOpen Access29 May 2019
Communications Biology

Volume: 2, P: 1-14
Interleukin-13 receptor alpha 2 cooperates with EGFRvIII signaling to promote glioblastoma multiforme

Interleukin-13 receptor alpha 2 is highly expressed in glioblastoma multiforme but its role in this malignancy is unclear. Here the authors show that this receptor interacts with mutant EGFR, stimulating its kinase activity, thus inducing proliferation.

Jennifer P. Newman
Grace Y. Wang
Paula Y. P. Lam
ResearchOpen Access04 Dec 2017
Nature Communications

Volume: 8, P: 1-17
Targeting proliferating tumor cells via the transcriptional control of therapeutic genes

I A W Ho
K M Hui
P Y P Lam
Research22 Jul 2005
Cancer Gene Therapy

Volume: 13, P: 44-52
Chimeric rabies SADB19-VSVg-pseudotyped lentiviral vectors mediate long-range retrograde transduction from the mouse spinal cord

L Schoderboeck
S Riad
S M Hughes
Research29 Jan 2015
Gene Therapy

Volume: 22, P: 357-364
Mutation of GATA3 in human breast tumors

Jerry Usary
Victor Llaca
Charles M Perou
Research13 Sept 2004
Oncogene

Volume: 23, P: 7669-7678
Melanocortin receptor-mediated effects on obesity are distributed over specific hypothalamic regions

M W A de Backer
S E la Fleur
R A H Adan
Research24 Aug 2010
International Journal of Obesity

Volume: 35, P: 629-641
Targeted expression of human folylpolyglutamate synthase for selective enhancement of methotrexate chemotherapy in osteosarcoma cells

K Bienemann
M S Staege
C M Kramm
Research16 Aug 2013
Cancer Gene Therapy

Volume: 20, P: 514-520
Mechanisms of distribution of mouse β-galactosidase in the adult GM1-gangliosidosis brain

M L D Broekman
L A Tierney
M Sena-Esteves
Research25 Sept 2008
Gene Therapy

Volume: 16, P: 303-308
Controlling brain tumor growth by intraventricular administration of an AAV vector encoding IFN-β

D H Meijer
C A Maguire
M Sena-Esteves
Research06 Feb 2009
Cancer Gene Therapy

Volume: 16, P: 664-671
Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease

Victoria J. McCurdy
Aime K. Johnson
Douglas R. Martin
Research03 Sept 2020
Gene Therapy

Volume: 28, P: 142-154
Erratum: Imaging gene delivery in a mouse model of congenital neuronal ceroid lipofuscinosis

L S Pike
B A Tannous
X O Breakefield
Amendments and Corrections08 Nov 2012
Gene Therapy

Volume: 19, P: 1121
Erratum: Gene therapy approach to FAP: in vivo influence of T119M in TTR deposition in a transgenic V30M mouse model

A R Batista
D Gianni
M J Saraiva
Amendments and Corrections04 Dec 2014
Gene Therapy

Volume: 21, P: 1065
Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease

V J McCurdy
H E Rockwell
D R Martin
Research04 Dec 2014
Gene Therapy

Volume: 22, P: 181-189
MS4A15 drives ferroptosis resistance through calcium-restricted lipid remodeling

Shan Xin
Constanze Mueller
Joel A. Schick
ResearchOpen Access18 Oct 2021
Cell Death & Differentiation

Volume: 29, P: 670-686
Gene therapy approach to FAP: in vivo influence of T119M in TTR deposition in a transgenic V30M mouse model

A R Batista
D Gianni
M J Saraiva
Research02 Oct 2014
Gene Therapy

Volume: 21, P: 1041-1050
Integration of active human β-galactosidase gene (100 kb) into genome using HSV/AAV amplicon vector

A Oehmig
M L Cortés
X O Breakefield
Research26 Apr 2007
Gene Therapy

Volume: 14, P: 1078-1091
Imaging gene delivery in a mouse model of congenital neuronal ceroid lipofuscinosis

L S Pike
B A Tannous
X O Breakefield
Research08 Sept 2011
Gene Therapy

Volume: 18, P: 1173-1178
Targeting HSV-1 virions for specific binding to epidermal growth factor receptor-vIII-bearing tumor cells

P Grandi
J Fernandez
X O Breakefield
Research28 May 2010
Cancer Gene Therapy

Volume: 17, P: 655-663
Imaging and therapy of experimental schwannomas using HSV amplicon vector-encoding apoptotic protein under Schwann cell promoter

S Prabhakar
G J Brenner
X O Breakefield
Research16 Oct 2009
Cancer Gene Therapy

Volume: 17, P: 266-274
A loss of FUS/TLS function leads to impaired cellular proliferation

C L Ward
K J Boggio
D A Bosco
ResearchOpen Access11 Dec 2014
Cell Death & Disease

Volume: 5, P: e1572
Suicidal gene therapy in an NF-κB-controlled tumor environment as monitored by a secreted blood reporter

C E Badr
J M Niers
B A Tannous
Research09 Dec 2010
Gene Therapy

Volume: 18, P: 445-451
Exosome-associated AAV vector as a robust and convenient neuroscience tool

E Hudry
C Martin
C A Maguire
Research02 Feb 2016
Gene Therapy

Volume: 23, P: 380-392

Search

Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy

Selective delivery of herpes virus vectors to experimental brain tumors using RMP-7

Enhanced green fluorescent protein fusion proteins of herpes simplex virus type 1 thymidine kinase and cytochrome P450 4B1: Applications for prodrug-activating gene therapy

Reactivated endogenous retroviruses promote protein aggregate spreading

Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial

AAV gene therapy for Tay-Sachs disease

Multimodal targeted high relaxivity thermosensitive liposome for in vivo imaging

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

Adeno-associated viral vectors for functional intravenous gene transfer throughout the non-human primate brain

GDNF gene therapy for alcohol use disorder in male non-human primates

Retrograde neuronal tracing with a deletion-mutant rabies virus

Therapeutic benefit of combining calorie-restricted ketogenic diet and glutamine targeting in late-stage experimental glioblastoma

Interleukin-13 receptor alpha 2 cooperates with EGFRvIII signaling to promote glioblastoma multiforme

Targeting proliferating tumor cells via the transcriptional control of therapeutic genes

Chimeric rabies SADB19-VSVg-pseudotyped lentiviral vectors mediate long-range retrograde transduction from the mouse spinal cord

Mutation of GATA3 in human breast tumors

Melanocortin receptor-mediated effects on obesity are distributed over specific hypothalamic regions

Targeted expression of human folylpolyglutamate synthase for selective enhancement of methotrexate chemotherapy in osteosarcoma cells

Mechanisms of distribution of mouse β-galactosidase in the adult GM1-gangliosidosis brain

Controlling brain tumor growth by intraventricular administration of an AAV vector encoding IFN-β

Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease

Erratum: Imaging gene delivery in a mouse model of congenital neuronal ceroid lipofuscinosis

Erratum: Gene therapy approach to FAP: in vivo influence of T119M in TTR deposition in a transgenic V30M mouse model

Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease

MS4A15 drives ferroptosis resistance through calcium-restricted lipid remodeling

Gene therapy approach to FAP: in vivo influence of T119M in TTR deposition in a transgenic V30M mouse model

Integration of active human β-galactosidase gene (100 kb) into genome using HSV/AAV amplicon vector

Imaging gene delivery in a mouse model of congenital neuronal ceroid lipofuscinosis

Targeting HSV-1 virions for specific binding to epidermal growth factor receptor-vIII-bearing tumor cells

Imaging and therapy of experimental schwannomas using HSV amplicon vector-encoding apoptotic protein under Schwann cell promoter

A loss of FUS/TLS function leads to impaired cellular proliferation

Suicidal gene therapy in an NF-κB-controlled tumor environment as monitored by a secreted blood reporter

Exosome-associated AAV vector as a robust and convenient neuroscience tool

Search

Quick links

Search

Filter By:

Search

Quick links