Spinal and bulbar muscular atrophy: From molecular pathogenesis to pharmacological intervention targeting skeletal muscle
Curr Opin Pharmacol. 2023 Aug;71:102394. doi: 10.1016/j.coph.2023.102394. Epub 2023 Jul 16.ABSTRACTThe clinical characteristics of SBMA, also known as Kennedy's disease (OMIM 313200), were initially documented by Dr. H Kawahara in the 18th century and a hundred years later by Dr. W. Kennedy. SBMA is a neuromuscular disease caused by expansions of a CAG microsatellite tandem repeat in exon 1 of the androgen receptor (AR) gene located on the X chromosome. These expansions result in the production of AR with an aberrantly expanded polyglutamine (polyQ) tract. In this review, we explore recent advancements in the significance ...
Source: Current Opinion in Pharmacology - July 18, 2023 Category: Drugs & Pharmacology Authors: Caterina Marchioretti Roberta Andreotti Emanuela Zuccaro Andrew P Lieberman Manuela Basso Maria Pennuto Source Type: research
Spinal and bulbar muscular atrophy: From molecular pathogenesis to pharmacological intervention targeting skeletal muscle
Curr Opin Pharmacol. 2023 Aug;71:102394. doi: 10.1016/j.coph.2023.102394. Epub 2023 Jul 16.ABSTRACTThe clinical characteristics of SBMA, also known as Kennedy's disease (OMIM 313200), were initially documented by Dr. H Kawahara in the 18th century and a hundred years later by Dr. W. Kennedy. SBMA is a neuromuscular disease caused by expansions of a CAG microsatellite tandem repeat in exon 1 of the androgen receptor (AR) gene located on the X chromosome. These expansions result in the production of AR with an aberrantly expanded polyglutamine (polyQ) tract. In this review, we explore recent advancements in the significance ...
Source: Current Opinion in Pharmacology - July 18, 2023 Category: Drugs & Pharmacology Authors: Caterina Marchioretti Roberta Andreotti Emanuela Zuccaro Andrew P Lieberman Manuela Basso Maria Pennuto Source Type: research
Spinal and bulbar muscular atrophy: From molecular pathogenesis to pharmacological intervention targeting skeletal muscle
Curr Opin Pharmacol. 2023 Aug;71:102394. doi: 10.1016/j.coph.2023.102394. Epub 2023 Jul 16.ABSTRACTThe clinical characteristics of SBMA, also known as Kennedy's disease (OMIM 313200), were initially documented by Dr. H Kawahara in the 18th century and a hundred years later by Dr. W. Kennedy. SBMA is a neuromuscular disease caused by expansions of a CAG microsatellite tandem repeat in exon 1 of the androgen receptor (AR) gene located on the X chromosome. These expansions result in the production of AR with an aberrantly expanded polyglutamine (polyQ) tract. In this review, we explore recent advancements in the significance ...
Source: Current Opinion in Pharmacology - July 18, 2023 Category: Drugs & Pharmacology Authors: Caterina Marchioretti Roberta Andreotti Emanuela Zuccaro Andrew P Lieberman Manuela Basso Maria Pennuto Source Type: research
Spinal and bulbar muscular atrophy: From molecular pathogenesis to pharmacological intervention targeting skeletal muscle
Curr Opin Pharmacol. 2023 Aug;71:102394. doi: 10.1016/j.coph.2023.102394. Epub 2023 Jul 16.ABSTRACTThe clinical characteristics of SBMA, also known as Kennedy's disease (OMIM 313200), were initially documented by Dr. H Kawahara in the 18th century and a hundred years later by Dr. W. Kennedy. SBMA is a neuromuscular disease caused by expansions of a CAG microsatellite tandem repeat in exon 1 of the androgen receptor (AR) gene located on the X chromosome. These expansions result in the production of AR with an aberrantly expanded polyglutamine (polyQ) tract. In this review, we explore recent advancements in the significance ...
Source: Current Opinion in Pharmacology - July 18, 2023 Category: Drugs & Pharmacology Authors: Caterina Marchioretti Roberta Andreotti Emanuela Zuccaro Andrew P Lieberman Manuela Basso Maria Pennuto Source Type: research
Spinal and bulbar muscular atrophy: From molecular pathogenesis to pharmacological intervention targeting skeletal muscle
Curr Opin Pharmacol. 2023 Jul 16;71:102394. doi: 10.1016/j.coph.2023.102394. Online ahead of print.ABSTRACTThe clinical characteristics of SBMA, also known as Kennedy's disease (OMIM 313200), were initially documented by Dr. H Kawahara in the 18th century and a hundred years later by Dr. W. Kennedy. SBMA is a neuromuscular disease caused by expansions of a CAG microsatellite tandem repeat in exon 1 of the androgen receptor (AR) gene located on the X chromosome. These expansions result in the production of AR with an aberrantly expanded polyglutamine (polyQ) tract. In this review, we explore recent advancements in the signi...
Source: Current Opinion in Pharmacology - July 18, 2023 Category: Drugs & Pharmacology Authors: Caterina Marchioretti Roberta Andreotti Emanuela Zuccaro Andrew P Lieberman Manuela Basso Maria Pennuto Source Type: research
Spinal and bulbar muscular atrophy: From molecular pathogenesis to pharmacological intervention targeting skeletal muscle
Curr Opin Pharmacol. 2023 Jul 16;71:102394. doi: 10.1016/j.coph.2023.102394. Online ahead of print.ABSTRACTThe clinical characteristics of SBMA, also known as Kennedy's disease (OMIM 313200), were initially documented by Dr. H Kawahara in the 18th century and a hundred years later by Dr. W. Kennedy. SBMA is a neuromuscular disease caused by expansions of a CAG microsatellite tandem repeat in exon 1 of the androgen receptor (AR) gene located on the X chromosome. These expansions result in the production of AR with an aberrantly expanded polyglutamine (polyQ) tract. In this review, we explore recent advancements in the signi...
Source: Current Opinion in Pharmacology - July 18, 2023 Category: Drugs & Pharmacology Authors: Caterina Marchioretti Roberta Andreotti Emanuela Zuccaro Andrew P Lieberman Manuela Basso Maria Pennuto Source Type: research
Spinal and bulbar muscular atrophy: From molecular pathogenesis to pharmacological intervention targeting skeletal muscle
Curr Opin Pharmacol. 2023 Jul 16;71:102394. doi: 10.1016/j.coph.2023.102394. Online ahead of print.ABSTRACTThe clinical characteristics of SBMA, also known as Kennedy's disease (OMIM 313200), were initially documented by Dr. H Kawahara in the 18th century and a hundred years later by Dr. W. Kennedy. SBMA is a neuromuscular disease caused by expansions of a CAG microsatellite tandem repeat in exon 1 of the androgen receptor (AR) gene located on the X chromosome. These expansions result in the production of AR with an aberrantly expanded polyglutamine (polyQ) tract. In this review, we explore recent advancements in the signi...
Source: Current Opinion in Pharmacology - July 18, 2023 Category: Drugs & Pharmacology Authors: Caterina Marchioretti Roberta Andreotti Emanuela Zuccaro Andrew P Lieberman Manuela Basso Maria Pennuto Source Type: research
Spinal and bulbar muscular atrophy: From molecular pathogenesis to pharmacological intervention targeting skeletal muscle
Curr Opin Pharmacol. 2023 Jul 16;71:102394. doi: 10.1016/j.coph.2023.102394. Online ahead of print.ABSTRACTThe clinical characteristics of SBMA, also known as Kennedy's disease (OMIM 313200), were initially documented by Dr. H Kawahara in the 18th century and a hundred years later by Dr. W. Kennedy. SBMA is a neuromuscular disease caused by expansions of a CAG microsatellite tandem repeat in exon 1 of the androgen receptor (AR) gene located on the X chromosome. These expansions result in the production of AR with an aberrantly expanded polyglutamine (polyQ) tract. In this review, we explore recent advancements in the signi...
Source: Current Opinion in Pharmacology - July 18, 2023 Category: Drugs & Pharmacology Authors: Caterina Marchioretti Roberta Andreotti Emanuela Zuccaro Andrew P Lieberman Manuela Basso Maria Pennuto Source Type: research
Spinal and bulbar muscular atrophy: From molecular pathogenesis to pharmacological intervention targeting skeletal muscle
Curr Opin Pharmacol. 2023 Jul 16;71:102394. doi: 10.1016/j.coph.2023.102394. Online ahead of print.ABSTRACTThe clinical characteristics of SBMA, also known as Kennedy's disease (OMIM 313200), were initially documented by Dr. H Kawahara in the 18th century and a hundred years later by Dr. W. Kennedy. SBMA is a neuromuscular disease caused by expansions of a CAG microsatellite tandem repeat in exon 1 of the androgen receptor (AR) gene located on the X chromosome. These expansions result in the production of AR with an aberrantly expanded polyglutamine (polyQ) tract. In this review, we explore recent advancements in the signi...
Source: Current Opinion in Pharmacology - July 18, 2023 Category: Drugs & Pharmacology Authors: Caterina Marchioretti Roberta Andreotti Emanuela Zuccaro Andrew P Lieberman Manuela Basso Maria Pennuto Source Type: research
Spinal and bulbar muscular atrophy: From molecular pathogenesis to pharmacological intervention targeting skeletal muscle
Curr Opin Pharmacol. 2023 Jul 16;71:102394. doi: 10.1016/j.coph.2023.102394. Online ahead of print.ABSTRACTThe clinical characteristics of SBMA, also known as Kennedy's disease (OMIM 313200), were initially documented by Dr. H Kawahara in the 18th century and a hundred years later by Dr. W. Kennedy. SBMA is a neuromuscular disease caused by expansions of a CAG microsatellite tandem repeat in exon 1 of the androgen receptor (AR) gene located on the X chromosome. These expansions result in the production of AR with an aberrantly expanded polyglutamine (polyQ) tract. In this review, we explore recent advancements in the signi...
Source: Current Opinion in Pharmacology - July 18, 2023 Category: Drugs & Pharmacology Authors: Caterina Marchioretti Roberta Andreotti Emanuela Zuccaro Andrew P Lieberman Manuela Basso Maria Pennuto Source Type: research
Spinal and bulbar muscular atrophy: From molecular pathogenesis to pharmacological intervention targeting skeletal muscle
Curr Opin Pharmacol. 2023 Jul 16;71:102394. doi: 10.1016/j.coph.2023.102394. Online ahead of print.ABSTRACTThe clinical characteristics of SBMA, also known as Kennedy's disease (OMIM 313200), were initially documented by Dr. H Kawahara in the 18th century and a hundred years later by Dr. W. Kennedy. SBMA is a neuromuscular disease caused by expansions of a CAG microsatellite tandem repeat in exon 1 of the androgen receptor (AR) gene located on the X chromosome. These expansions result in the production of AR with an aberrantly expanded polyglutamine (polyQ) tract. In this review, we explore recent advancements in the signi...
Source: Current Opinion in Pharmacology - July 18, 2023 Category: Drugs & Pharmacology Authors: Caterina Marchioretti Roberta Andreotti Emanuela Zuccaro Andrew P Lieberman Manuela Basso Maria Pennuto Source Type: research
Spinal and bulbar muscular atrophy: From molecular pathogenesis to pharmacological intervention targeting skeletal muscle
Curr Opin Pharmacol. 2023 Jul 16;71:102394. doi: 10.1016/j.coph.2023.102394. Online ahead of print.ABSTRACTThe clinical characteristics of SBMA, also known as Kennedy's disease (OMIM 313200), were initially documented by Dr. H Kawahara in the 18th century and a hundred years later by Dr. W. Kennedy. SBMA is a neuromuscular disease caused by expansions of a CAG microsatellite tandem repeat in exon 1 of the androgen receptor (AR) gene located on the X chromosome. These expansions result in the production of AR with an aberrantly expanded polyglutamine (polyQ) tract. In this review, we explore recent advancements in the signi...
Source: Current Opinion in Pharmacology - July 18, 2023 Category: Drugs & Pharmacology Authors: Caterina Marchioretti Roberta Andreotti Emanuela Zuccaro Andrew P Lieberman Manuela Basso Maria Pennuto Source Type: research
Spinal and bulbar muscular atrophy: From molecular pathogenesis to pharmacological intervention targeting skeletal muscle
Curr Opin Pharmacol. 2023 Jul 16;71:102394. doi: 10.1016/j.coph.2023.102394. Online ahead of print.ABSTRACTThe clinical characteristics of SBMA, also known as Kennedy's disease (OMIM 313200), were initially documented by Dr. H Kawahara in the 18th century and a hundred years later by Dr. W. Kennedy. SBMA is a neuromuscular disease caused by expansions of a CAG microsatellite tandem repeat in exon 1 of the androgen receptor (AR) gene located on the X chromosome. These expansions result in the production of AR with an aberrantly expanded polyglutamine (polyQ) tract. In this review, we explore recent advancements in the signi...
Source: Current Opinion in Pharmacology - July 18, 2023 Category: Drugs & Pharmacology Authors: Caterina Marchioretti Roberta Andreotti Emanuela Zuccaro Andrew P Lieberman Manuela Basso Maria Pennuto Source Type: research
Targeting cAMP signaling compartments in iPSC-derived models of cardiovascular disease
Curr Opin Pharmacol. 2023 Jul 13;71:102392. doi: 10.1016/j.coph.2023.102392. Online ahead of print.ABSTRACTAdenosine 3',5'-cyclic monophosphate (cAMP) acts as a second messenger that is involved in the regulation of a plethora of processes. The activation of cAMP signaling in defined compartments is critical for cells to respond to an extracellular stimulus in a specific manner. Rapid advances in the field of human induced pluripotent stem cells (iPSCs) reflect their great potential for cardiovascular disease modeling, drug screening, regenerative and precision medicine. This review discusses cAMP signaling in iPSC-derived...
Source: Current Opinion in Pharmacology - July 15, 2023 Category: Drugs & Pharmacology Authors: Tiannan Liu Enno Klussmann Source Type: research
Targeting cAMP signaling compartments in iPSC-derived models of cardiovascular disease
Curr Opin Pharmacol. 2023 Jul 13;71:102392. doi: 10.1016/j.coph.2023.102392. Online ahead of print.ABSTRACTAdenosine 3',5'-cyclic monophosphate (cAMP) acts as a second messenger that is involved in the regulation of a plethora of processes. The activation of cAMP signaling in defined compartments is critical for cells to respond to an extracellular stimulus in a specific manner. Rapid advances in the field of human induced pluripotent stem cells (iPSCs) reflect their great potential for cardiovascular disease modeling, drug screening, regenerative and precision medicine. This review discusses cAMP signaling in iPSC-derived...
Source: Current Opinion in Pharmacology - July 15, 2023 Category: Drugs & Pharmacology Authors: Tiannan Liu Enno Klussmann Source Type: research
