Recent Articles2024-12-10T01:40:29+00:00
6August, 2024

ALS: From Genetic Complexity to Treatment Challenges and Advances

Categories: Perspectives on Current Science|

Amyotrophic Lateral Sclerosis (ALS), commonly called Lou Gehrig's disease, is a neurodegenerative disorder of the motor neurons that causes progressive muscle weakness and respiratory failure (1). ALS is the most common motor disease among adults (2), affecting around 60,000 people in the U.S. and Europe. Life expectancy for those ...

16July, 2024

Inherited Retinal Disorder: In Vivo CRISPR Therapy Provides Vision Improvements

Categories: Perspectives on Current Science|

Inherited retinal disorders (IRDs) are the leading cause of visual impairment, affecting approximately 1 in 2,000 people of all ages worldwide (1). There are many types of IRDs, each caused by a gene variant that affects how the retina functions, with one of the most severe being Leber Congenital ...

24June, 2024

Angelman Syndrome Treatment Efforts and a Novel Prenatal Strategy

Categories: Perspectives on Current Science|

Imagine finding out your child may never walk, talk, feed, or dress themselves. Never have a job, get married, or have children. Never live independently. This long list of milestones never to be met is the experience for many parents with children diagnosed with Angelman syndrome (AS), a rare, ...

7May, 2024

Hope of New Treatments for Chronic Liver Disease MASH (NASH)

Categories: Perspectives on Current Science|

In the early stages of Metabolic Dysfunction-Associated Steatohepatitis (MASH), many patients are asymptomatic, though some may feel weak or fatigued or have an ache in their upper right abdomen. While lifestyle changes can help patients diagnosed early enough, more advanced stages of the disease have fewer options. However, recent ...

16April, 2024

RNA Single-base Editing Therapy that Treats Genetic Lung and Liver Disease Entered Clinical Trials

Categories: Perspectives on Current Science|

When Peggy's mom was in her forties, she started to have trouble breathing. Although she had never smoked, she had been exposed second-hand earlier in her life. Eventually, her mother ended up at the Mayo Clinic, where she was the 36th patient to be diagnosed with Alpha-1 Antitrypsin Deficiency ...

1April, 2024

Jennifer Doudna – Seeking to Improve the World with CRISPR

Categories: Perspectives on Current Science|

At 10:53 p.m. on Oct. 7, Jennifer Doudna woke to a buzzing sound. Picking up her phone — which she noticed had multiple missed calls and messages — she was greeted by the voice of Nature journalist Heidi Ledford. The day before, Doudna had been in an all-day meeting; ...

14March, 2024

FDA Approved the First CRISPR Treatment – Potential Cure for People with Sickle Cell Disease

Categories: Perspectives on Current Science|

Victoria Gray was diagnosed with sickle cell disease (SCD) when she was just three months old; since then, episodes of pain and frequent hospitalizations have been a part of her life. Many of her dreams seemed like far-off impossibilities when the smallest things, like changing weather, would put her ...

21February, 2024

Follow-up Data Confirms That Inclisiran Provides Long-Term Reduction of LDL-Cholesterol

Categories: Perspectives on Current Science|

Cardiovascular disease is the leading cause of death in the United States, exceeding all types of cancer, unintentional injury, and stroke combined. However, updated data from Novartis's open-label trial shows promising long-term results for inclisiran (Leqvio), the first and only small interfering RNA (siRNA) therapy to reduce low-density lipoprotein ...

9February, 2024

The First FDA Approval for a GalNAc-conjugated ASO

Categories: Perspectives on Current Science|

It starts with a pins-and-needle sensation in your feet, or maybe your gastrointestinal tract has become easily irritated, or you've started to lose weight or develop heart problems. Hereditary ATTR amyloidosis can present differently among patients and with symptoms found in far more common diseases, so it is often ...

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