肌肉萎缩症协会授予近 500 万美元的新拨款，用于资助肌肉萎缩症及相关肌肉疾病的研究

肌肉营养不良协会 (MDA) 已授予近两打新拨款，用于资助各种形式的肌肉营养不良的研究，包括杜氏肌营养不良症和肢带肌营养不良症，以及相关的肌肉疾病。

The Muscular Dystrophy Association (MDA) has awarded nearly two dozen new grants to fund research into various forms of muscular dystrophy, including Duchenne and limb-girdle, as well as related muscle diseases.

肌肉营养不良协会 (MDA) 已授予近两打新拨款，用于资助各种形式的肌肉营养不良的研究，包括杜氏肌营养不良症和肢带肌营养不良症，以及相关的肌肉疾病。

Altogether, the funding totals more than $5 million across 21 grants, according to a press release from the nonprofit.

根据该非营利组织的新闻稿，21 项赠款的资金总额超过 500 万美元。

“The Muscular Dystrophy Association is proud to continue driving critical research that brings hope and tangible progress to the neuromuscular community,” said Donald S. Wood, PhD, president and CEO of the MDA. “These projects reflect our dedication to the people we serve; we are eager to see how they will fuel clinical and scientific progress and, ultimately, lead to new treatments.”

“肌肉营养不良协会很自豪能够继续推动关键研究，为神经肌肉界带来希望和切实进展，”MDA 总裁兼首席执行官唐纳德·S·伍德博士说。 “这些项目体现了我们对我们所服务的人们的奉献精神；我们渴望看到它们将如何推动临床和科学进步，并最终带来新的治疗方法。”

There are 14 research grants in all, as well as seven development awards for fellowship trainees. The MDA partnered with two other nonprofits on three collaboration grants — one for a University of California Los Angeles researcher seeking to develop a gene therapy for a type of limb-girdle muscular dystrophy (LGMD).

共有 14 项研究资助，以及 7 项针对实习生的发展奖。 MDA 与另外两个非营利组织合作提供了三项合作资助，其中一项资助给加州大学洛杉矶分校的一名研究人员，旨在开发一种针对肢带型肌营养不良症 (LGMD) 的基因疗法。

Over $5M in funding awarded across 21 MDA grants

21 项 MDA 拨款超过 500 万美元

The newly awarded funding includes several research grants to well-established scientists. For example, Jyoti Jaiswal, PhD, a professor at Children’s National Research and Innovation, a hospital in Washington, D.C., won funding for his work to prevent muscle cell death and damage in Duchenne muscular dystrophy, known as DMD.

新授予的资金包括向知名科学家提供的几项研究资助。例如，华盛顿特区儿童国家研究与创新医院的教授 Jyoti Jaiswal 博士因其预防杜氏肌营养不良症（DMD）肌肉细胞死亡和损伤的工作而获得了资助。

DMD is caused by gene mutations that lead to a lack of dystrophin — a protein that normally acts like a shock absorber in muscle cells to limit damage during muscle contractions. Without dystrophin, muscles accumulate more wear and tear damage over time, ultimately driving disease symptoms. Jaiswal’s project will explore a therapy that targets fat-like molecules to stabilize muscle cell membranes, with the goal of reducing muscle damage and improving function in DMD.

DMD 是由导致肌营养不良蛋白缺乏的基因突变引起的，这种蛋白通常在肌肉细胞中起到减震器的作用，以限制肌肉收缩期间的损伤。如果没有肌营养不良蛋白，肌肉会随着时间的推移积累更多的磨损和撕裂损伤，最终导致疾病症状。 Jaiswal 的项目将探索一种针对脂肪类分子来稳定肌肉细胞膜的疗法，其目标是减少肌肉损伤并改善 DMD 的功能。

Another of the newly funded projects will explore the use of gene-editing technologies to treat LGMD, a group of genetic disorders characterized by muscle wasting in the shoulders and hips. That project, led by Scot Wolfe, PhD, of the University of Massachusetts Chan Medical School, will be testing gene editing to correct mutations that cause LGMD type 2G/R7. Wolfe is using stem cells, a mouse model, and other approaches to develop a platform that could also be expanded to treat other types of LGMD.

另一个新资助的项目将探索使用基因编辑技术来治疗 LGMD，这是一组以肩膀和臀部肌肉萎缩为特征的遗传性疾病。该项目由马萨诸塞大学陈医学院的 Scot Wolfe 博士领导，将测试基因编辑以纠正导致 LGMD 2G/R7 型的突变。 Wolfe 正在使用干细胞、小鼠模型和其他方法来开发一个平台，该平台也可以扩展到治疗其他类型的 LGMD。

A separate project focused on LGMD is being jointly funded by the MDA and the Coalition to Cure Calpain 3. That project, led by Melissa Spencer, PhD, co-director of the Center for Duchenne Muscular Dystrophy at UCLA, aims to develop a gene therapy for LGMD type 2A/R1. This type of LGMD is caused by mutations in the CAPN3 gene; the project is focused on finding effective ways to deliver a healthy version of this gene to muscle cells.

MDA 和治愈 Calpain 3 联盟联合资助了一个专注于 LGMD 的独立项目。该项目由加州大学洛杉矶分校杜氏肌营养不良症中心联席主任 Melissa Spencer 博士领导，旨在开发一种基因疗法适用于 LGMD 2A/R1 型。这种类型的 LGMD 是由 CAPN3 基因突变引起的；该项目的重点是寻找有效的方法将该基因的健康版本传递给肌肉细胞。

“We are excited to support Dr. Spencer’s groundbreaking work in advancing gene therapy for LGMD2A/R1,” Jennifer R. Levy, PhD, scientific director of the Coalition to Cure Calpain 3, said in a separate press release. “By funding this project, we are not only pushing the boundaries of what’s possible in genetic research but also reinforcing our commitment to finding life-altering treatments for those affected by this rare condition. This collaboration with MDA will enable us to accelerate progress and bring hope to the LGMD2A/R1 community.”

“我们很高兴能够支持 Spencer 博士在推进 LGMD2A/R1 基因治疗方面的开创性工作，”治愈 Calpain 3 联盟的科学主任 Jennifer R. Levy 博士在另一份新闻稿中表示。 “通过资助这个项目，我们不仅突破了基因研究的可能界限，而且加强了我们为受这种罕见疾病影响的人们寻找改变生活的治疗方法的承诺。与 MDA 的合作将使我们能够加快进展，并为 LGMD2A/R1 社区带来希望。”

Spencer said the new funding “will allow us to refine our gene therapy approach and move closer to a treatment that could significantly improve the quality of life for people living with LGMD 2A/R1.”

Spencer 表示，新的资金“将使我们能够完善基因治疗方法，并更接近于能够显着改善 LGMD 2A/R1 患者生活质量的治疗方法。”

According to Sharon Hesterlee, PhD, the MDA’s chief research officer, this work by Spencer “represents a major step forward in gene therapy” for this type of LGMD.

MDA 首席研究官 Sharon Hesterlee 博士表示，Spencer 的这项工作“代表了此类 LGMD 基因治疗的重大进步”。

“Our partnership with C3 highlights the importance of collaboration in advancing research and finding potential cures for neuromuscular diseases,” Hesterlee said.

“我们与 C3 的合作凸显了合作在推进研究和寻找神经肌肉疾病潜在治疗方法方面的重要性，”Hesterlee 说。

Other projects funded by the MDA in this round will focus on neuromuscular conditions like spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS).

本轮 MDA 资助的其他项目将重点关注脊髓性肌萎缩症 (SMA) 和肌萎缩侧索硬化症 (ALS) 等神经肌肉疾病。

The MDA also has awarded more than a half-dozen grants to support early-career researchers studying muscular dystrophy and related disorders, with project goals ranging from making gene therapy safer to evaluating the effects of hormones in specific types of muscular dystrophy

MDA 还授予了超过六项拨款，以支持研究肌营养不良症和相关疾病的早期职业研究人员，项目目标包括使基因治疗更安全，到评估激素对特定类型肌营养不良症的影响

“We are excited to award these new grants to researchers whose work has the potential to bring us closer to disease-modifying treatments for neuromuscular diseases,” Hesterlee said. “These grants not only support established researchers but also nurture the next generation of scientists who will continue the fight against these debilitating diseases.”

海斯特利说：“我们很高兴将这些新的资助授予研究人员，他们的工作有可能使我们更接近神经肌肉疾病的疾病缓解治疗。” “这些赠款不仅支持现有的研究人员，还培养下一代科学家，他们将继续与这些使人衰弱的疾病作斗争。”

The MDA’s investment to date in neuromuscular disease research exceeds $1 billion, according to the association.

据该协会称，MDA 迄今为止在神经肌肉疾病研究方面的投资超过 10 亿美元。