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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
1986 1
1987 2
1988 2
1989 12
1990 24
1991 36
1992 55
1993 108
1994 154
1995 223
1996 309
1997 446
1998 475
1999 613
2000 871
2001 1014
2002 1131
2003 1301
2004 1298
2005 1413
2006 1367
2007 1281
2008 1293
2009 1148
2010 1138
2011 1201
2012 1104
2013 875
2014 845
2015 1037
2016 1015
2017 1029
2018 972
2019 379
2020 3
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22,446 results
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Page 1
AAV: An Overview of Unanswered Questions
Berns KI and Muzyczka N. Hum Gene Ther 2017 - Review. PMID 28335618 Free PMC article.
Some of these are touched on in the six review articles in this issue of Human Gene Therapy....
Some of these are touched on in the six review articles in this issue of Human Gene Therapy....
In vivo genome editing via the HITI method as a tool for gene therapy.
Suzuki K and Izpisua Belmonte JC. J Hum Genet 2018 - Review. PMID 29215090
Using genome-editing technologies to correct specific mutations represents a potentially transformative new approach for treating genetic disorders. ...This poses a significant barrier for developing therapeutic strategies to treat a broad range of devastating genetic disorders. ...
Using genome-editing technologies to correct specific mutations represents a potentially transformative new approach for treating genetic
Treatment Advances in Spinal Muscular Atrophy
Bharucha-Goebel D and Kaufmann P. Curr Neurol Neurosci Rep 2017 - Review. PMID 28983837 Free PMC article.
PURPOSE OF REVIEW: Spinal muscular atrophy (SMA) is a genetic disorder of motor neurons in the anterior horns of the spinal cord and brainstem that results in muscle atrophy and weakness. ...There are several other promising therapeutics in different stages of development, based on approaches such as neuroprotection, or gene therapy....
PURPOSE OF REVIEW: Spinal muscular atrophy (SMA) is a genetic disorder of motor neurons in the anterior horns of the spinal cord and …
Gene Therapy Now?
Pederson T. FASEB J 2018. PMID 29592791
Gene Therapy for Neurodegenerative Diseases.
Sudhakar V and Richardson RM. Neurotherapeutics 2019 - Review. PMID 30542906 Free PMC article.
Gene therapy has the potential to provide therapeutic benefit to millions of people with neurodegenerative diseases through several means, including direct correction of pathogenic mechanisms, neuroprotection, neurorestoration, and symptom control. ...The gene therapy field has recently entered a new technological era, in which interventional MRI-guided convection-enhanced delivery (iMRI-CED) is the gold standard for verifying accurate vector delivery in real time. ...
Gene therapy has the potential to provide therapeutic benefit to millions of people with neurodegenerative diseases through several m …
Clinical advances in gene therapy updates on clinical trials of gene therapy in haemophilia.
Peyvandi F and Garagiola I. Haemophilia 2019 - Review. PMID 31282050
Gene therapy is rapidly becoming a new therapeutic strategy for haemophilia A and B treatment. In the 1990s, studies in animal models showed that adeno-associated vectors (AAV) exhibited an efficient expression of factor IX (FIX). ...Clinical trials with adeno-associated vectors have documented a significant success for haemophilia gene therapy demonstrating potential to transform haemophilia treatment offering hope for a long-term expression....
Gene therapy is rapidly becoming a new therapeutic strategy for haemophilia A and B treatment. In the 1990s, studies in animal models …
Advances in CRISPR/Cas9 Technology for in Vivo Translation
Çiçek YA, et al. Biol Pharm Bull 2019 - Review. PMID 30828060 Free article.
The complexity of in vivo delivery is furthered by the number of potential delivery methods, the different forms in which CRISPR can be delivered as a therapeutic, and the disease target and tissue type in question. ...
The complexity of in vivo delivery is furthered by the number of potential delivery methods, the different forms in which CRISPR can …
Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches.
Fraldi A, et al. Ital J Pediatr 2018 - Review. PMID 30442177 Free PMC article.
The current therapeutic strategies of enzyme replacement therapy and allogeneic hematopoietic stem cell transplantation have been reported to reduce patient morbidity and to improve their quality of life, but they are associated with persistence of residual disease burden, in particular at the neurocognitive and musculoskeletal levels. ...Gene therapy (GT) strategies aimed at correcting the genetic defect in patient cells could represent a significant improvement for the treatment of MPS when compared with conventional approaches. ...
The current therapeutic strategies of enzyme replacement therapy and allogeneic hematopoietic stem cell transplantation have been rep …
Adeno Associated Virus (AAV) as a Tool for Clinical and Experimental Delivery of Target Genes into the Mammalian Retina
Hori T, et al. Biol Pharm Bull 2019 - Review. PMID 30828065 Free article.
With an increasing number of identified causative genes, the widespread use of gene therapy is quickly becoming feasible. Once a target gene is selected, it is important to have a cell delivery method that is both specific and efficient. ...
With an increasing number of identified causative genes, the widespread use of gene therapy is quickly becoming feasible. Once a targ …
Gene Therapy in the Nervous System: Failures and Successes.
Chandran JS, et al. Adv Exp Med Biol 2017 - Review. PMID 28840561
Genetic disorders, caused by deleterious changes in the DNA sequence away from the normal genomic sequence, affect millions of people worldwide. Gene therapy as a treatment option for patients is an attractive proposition due to its conceptual simplicity. In principle, gene therapy involves correcting the genetic disorder by either restoring a normal functioning copy of a gene or reducing the toxicity arising from a mutated gene. ...
Genetic disorders, caused by deleterious changes in the DNA sequence away from the normal genomic sequence, affect millions of people
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