(Q90677465)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30065971%20AND%20SRC:MED&resulttype=core&format=json

15 April 2020

title

How will the field of gene therapy survive its success? (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30065971%20AND%20SRC:MED&resulttype=core&format=json

15 April 2020

author name string

William F Kaemmerer

series ordinal

1

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30065971%20AND%20SRC:MED&resulttype=core&format=json

15 April 2020

publication date

24 May 2018

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30065971%20AND%20SRC:MED&resulttype=core&format=json

Bioengineering & Translational Medicine

15 April 2020

published in

1 reference

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15 April 2020

volume

3

1 reference

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15 April 2020

issue

2

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15 April 2020

page(s)

166-177

1 reference

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Zinc finger peptides for the regulation of gene expression

15 April 2020

cites work

1 reference

12 December 2020

Short hairpin RNAs (shRNAs) induce sequence-specific silencing in mammalian cells

1 reference

12 December 2020

Gene therapy. Second child in French trial is found to have leukemia

1 reference

12 December 2020

Inducible systemic RNA silencing in Caenorhabditis elegans

1 reference

12 December 2020

Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

1 reference

12 December 2020

Towards therapeutic applications of engineered zinc finger proteins

1 reference

12 December 2020

Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.

1 reference

12 December 2020

Scalable generation of high-titer recombinant adeno-associated virus type 5 in insect cells

1 reference

12 December 2020

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

1 reference

12 December 2020

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

1 reference

12 December 2020

Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential

1 reference

12 December 2020

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

1 reference

12 December 2020

Striatal readministration of rAAV vectors reveals an immune response against AAV2 capsids that can be circumvented.

1 reference

12 December 2020

Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons

1 reference

12 December 2020

A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells

1 reference

12 December 2020

The neonatal blood-brain barrier is functionally effective, and immaturity does not explain differential targeting of AAV9.

1 reference

12 December 2020

Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs

1 reference

12 December 2020

AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial

1 reference

12 December 2020

Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates

1 reference

12 December 2020

Introduction to viral vectors

1 reference

12 December 2020

Duchenne muscular dystrophy gene therapy: Lost in translation?

1 reference

12 December 2020

Viral vectors for gene delivery to the central nervous system

1 reference

12 December 2020

Widespread suppression of huntingtin with convection-enhanced delivery of siRNA

1 reference

12 December 2020

Comparison of gene transfer to the murine liver following intraperitoneal and intraportal delivery of hepatotropic AAV pseudo-serotypes.

1 reference

12 December 2020

We still have a long way to go to effectively deliver genes!

1 reference

12 December 2020

Production and characterization of novel recombinant adeno-associated virus replicative-form genomes: a eukaryotic source of DNA for gene transfer

1 reference

12 December 2020

Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.

1 reference

12 December 2020

Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease

1 reference

12 December 2020

Non-viral vectors for gene-based therapy

1 reference

12 December 2020

A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.

1 reference

12 December 2020

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

1 reference

12 December 2020

Non viral vectors in gene therapy- an overview

1 reference

12 December 2020

$1-million price tag set for Glybera gene therapy

1 reference

12 December 2020

Scalable downstream strategies for purification of recombinant adeno- associated virus vectors in light of the properties

1 reference

12 December 2020

Re-examination of regulatory opinions in Europe: possible contribution for the approval of the first gene therapy product Glybera

1 reference

12 December 2020

Onset Time and Durability of Huntingtin Suppression in Rhesus Putamen After Direct Infusion of Antihuntingtin siRNA.

1 reference

12 December 2020

Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus.

1 reference

12 December 2020

Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease

1 reference

12 December 2020

Antisense oligonucleotide-directed inhibition of nonsense-mediated mRNA decay

1 reference

12 December 2020

Long-Term Safety of Patients with Parkinson's Disease Receiving rAAV2-Neurturin (CERE-120) Gene Transfer

1 reference

12 December 2020

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

1 reference

12 December 2020

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.

1 reference

12 December 2020

Tet-On Systems For Doxycycline-inducible Gene Expression.

1 reference

12 December 2020

Current status of non-viral gene therapy for CNS disorders

1 reference

12 December 2020

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

1 reference

12 December 2020

Viral Vectors: The Road to Reducing Genotoxicity

1 reference

12 December 2020

Alternative designs for clinical trials in rare diseases

1 reference

12 December 2020

Clinical Trial Design for Alpha-1 Antitrypsin Deficiency: A Model for Rare Diseases

1 reference

12 December 2020

The Self-Inactivating KamiCas9 System for the Editing of CNS Disease Genes

1 reference

12 December 2020

Intravenous administration of the adeno-associated virus-PHP.B capsid fails to upregulate transduction efficiency in the marmoset brain.

1 reference

12 December 2020

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

1 reference

12 December 2020

AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.

1 reference

12 December 2020

AAV gene delivery to the spinal cord: serotypes, methods, candidate diseases, and clinical trials

1 reference

12 December 2020

Severe Toxicity in Nonhuman Primates and Piglets with Systemic High-Dose Administration of Adeno-Associated Virus Serotype 9-Like Vectors: Putting Patients First.

1 reference

12 December 2020

Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an AAV vector expressing human SMN.

1 reference

12 December 2020

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice.

1 reference

12 December 2020

Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety.

1 reference

12 December 2020

Pharmacology of Recombinant Adeno-associated Virus Production.

1 reference

12 December 2020

Identifiers

DOI

10.1002/BTM2.10090

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30065971%20AND%20SRC:MED&resulttype=core&format=json

15 April 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:30065971%20AND%20SRC:MED&resulttype=core&format=json

15 April 2020

1 reference