| 1 |
35046408 | SUPT4H1-edited stem cell therapy rescues neuronal dysfunction in a mouse model for Huntington's disease. | 2022 Jan 19 |
1 |
| 2 |
35254632 | Antisense Oligonucleotide Therapy: From Design to the Huntington Disease Clinic. | 2022 Mar |
1 |
| 3 |
33731741 | A high-throughput screening to identify small molecules that suppress huntingtin promoter activity or activate huntingtin-antisense promoter activity. | 2021 Mar 17 |
1 |
| 4 |
33889127 | Gene Therapy in Movement Disorders: A Systematic Review of Ongoing and Completed Clinical Trials. | 2021 |
3 |
| 5 |
34520257 | Oligonucleotides Targeting DNA Repeats Downregulate Huntingtin Gene Expression in Huntington's Patient-Derived Neural Model System. | 2021 Dec |
2 |
| 6 |
34658796 | Mislocalization of Nucleocytoplasmic Transport Proteins in Human Huntington's Disease PSC-Derived Striatal Neurons. | 2021 |
2 |
| 7 |
34899193 | Innovative Therapeutic Approaches for Huntington's Disease: From Nucleic Acids to GPCR-Targeting Small Molecules. | 2021 |
1 |
| 8 |
34918046 | Therapeutic reversal of Huntington's disease by in vivo self-assembled siRNAs. | 2021 Dec 16 |
2 |
| 9 |
32202594 | Huntingtin-Lowering Therapies for Huntington Disease: A Review of the Evidence of Potential Benefits and Risks. | 2020 Jun 1 |
1 |
| 10 |
32245050 | Huntingtin Lowering Strategies. | 2020 Mar 20 |
1 |
| 11 |
32796930 | Huntington disease: new insights into molecular pathogenesis and therapeutic opportunities. | 2020 Oct |
1 |
| 12 |
30616867 | Recent advances in the therapeutic development for Huntington disease. | 2019 Feb |
1 |
| 13 |
31059641 | Targeting Huntingtin Expression in Patients with Huntington's Disease. | 2019 Jun 13 |
2 |
| 14 |
31184975 | Lowering Mutant Huntingtin Using Tricyclo-DNA Antisense Oligonucleotides As a Therapeutic Approach for Huntington's Disease. | 2019 Oct |
3 |
| 15 |
31381521 | Characterization of Neurodevelopmental Abnormalities in iPSC-Derived Striatal Cultures from Patients with Huntington's Disease. | 2019 |
1 |
| 16 |
29535594 | Precise Excision of the CAG Tract from the Huntingtin Gene by Cas9 Nickases. | 2018 |
1 |
| 17 |
29620999 | Dose-Dependent Lowering of Mutant Huntingtin Using Antisense Oligonucleotides in Huntington Disease Patients. | 2018 Apr |
2 |
| 18 |
28334749 | CTG repeat-targeting oligonucleotides for down-regulating Huntingtin expression. | 2017 May 19 |
2 |
| 19 |
28920889 | Therapies targeting DNA and RNA in Huntington's disease. | 2017 Oct |
2 |
| 20 |
29209494 | RNAi mechanisms in Huntington's disease therapy: siRNA versus shRNA. | 2017 |
1 |
| 21 |
26201449 | Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry. | 2015 Nov |
2 |
| 22 |
26222265 | Allele-Selective Inhibition of Mutant Huntingtin with 2-Thio- and C5- Triazolylphenyl-Deoxythymidine-Modified Antisense Oligonucleotides. | 2015 Oct |
1 |
| 23 |
25155142 | Targets for future clinical trials in Huntington's disease: what's in the pipeline? | 2014 Sep 15 |
1 |
| 24 |
25164989 | Huntingtin-lowering strategies in Huntington's disease: antisense oligonucleotides, small RNAs, and gene editing. | 2014 Sep 15 |
1 |
| 25 |
25207939 | Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients. | 2014 |
1 |
| 26 |
26865404 | Effect of 2'-O-methyl/thiophosphonoacetate-modified antisense oligonucleotides on huntingtin expression in patient-derived cells. | 2014 Dec 15 |
1 |
| 27 |
23963702 | Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS. | 2013 Nov |
2 |
| 28 |
26307259 | Red/ET recombination with chimeric oligonucleotides allows rapid generation of BAC transgenes harboring full-length or truncated huntingtin cDNA. | 2012 Jul 1 |
1 |
| 29 |
21909428 | Targeting several CAG expansion diseases by a single antisense oligonucleotide. | 2011 |
2 |
| 30 |
21971427 | Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin. | 2011 Dec |
1 |
| 31 |
21028906 | Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat. | 2010 Nov 30 |
3 |
| 32 |
20082975 | Huntington disease: pathogenesis, biomarkers, and approaches to experimental therapeutics. | 2009 Dec |
1 |
| 33 |
10814708 | Decreased expression of striatal signaling genes in a mouse model of Huntington's disease. | 2000 May 22 |
1 |